Genome-Scale CRISPRi Atlas Maps Gene Function Across Human iPSCs

A new genome-scale atlas is offering an unprecedented look at how individual genes shape the transcriptional landscape of human induced pluripotent stem cells (iPSCs). Published in Nature Biotechnology, the resource catalogs the effects of perturbing 11,692 expressed genes across more than 2.5 million single cells, creating a reference framework for understanding how pluripotent identity is maintained and regulated.

The study is titled, “A genome-scale CRISPRi perturbation atlas of human induced pluripotent stem cells.”

Human iPSCs can differentiate into virtually any cell type, yet the functions of most genes within this state remain poorly understood. Prashant Mali, PhD, senior author and professor of bioengineering at UC San Diego, said the team set out to fill that gap by systematically switching off genes one by one using CRISPR interference (CRISPRi) and measuring the resulting transcriptome-wide changes. “The result is a kind of reference atlas; it’s a way to look up what perturbing almost any gene does to a stem cell’s behavior, measured here as the impact on its whole transcriptome,” Mali said.

The dataset captures how gene perturbations cluster into shared molecular signatures, revealing functional relationships among protein complexes, metabolic pathways, and self-renewal genes. By correlating transcriptional phenotypes across thousands of perturbations, the researchers reconstructed a map of the pluripotent state that recapitulates known regulatory modules while surfacing previously unrecognized ones.

Exploring the atlas led the team to identify new regulators of stem cell biology. They uncovered ZBTB41 as a metabolic factor and RNF7 as a contributor to pluripotency regulation, validating both through metabolic tracing, immunofluorescence, and protein–protein interaction assays. The resource also enabled a genome-scale screen of A‑to‑I RNA editing modulators, revealing DBR1 as a potent regulator of adenosine-to-inosine conversion.

Co-first author Yesh Doctor, a bioengineering PhD student in Mali’s lab, described the atlas as a “hypothesis engine” for stem cell researchers. Instead of running thousands of perturbation experiments, scientists can now query the open-access map to identify candidate genes involved in differentiation, metabolism, or disease-relevant pathways. “Scientists can use it to look up the functions of genes and build hypotheses on them instead of having to run the experiments themselves,” Doctor said.

Beyond basic biology, the team sees the atlas as a foundation for computational modeling. The scale and consistency of the dataset make it well suited for training AI systems aimed at predicting genotype–phenotype relationships. “These comprehensive, genome-scale screens enable generation of reference maps that are not just invaluable for basic science discovery, but also an important resource for powering future computational and AI tools for genotype-phenotype prediction,” Mali said.

The open-access atlas is available here, providing a new reference point for understanding how genes shape human stem cell identity and offering a tool for virtual disease modeling and target discovery.

The post Genome-Scale CRISPRi Atlas Maps Gene Function Across Human iPSCs appeared first on GEN – Genetic Engineering and Biotechnology News.

A Web-Based Self-Management Intervention for Return-to-Work Among Persons With Common Mental Disorders on Sick Leave: Case Study of mWorks

Background: mWorks is a co-designed, web-based self-management intervention developed to empower persons with common mental disorders who are on sick leave during the return-to-work process. However, limited knowledge of how mWorks is delivered and engaged with in real-world settings constrains further development and implementation. In line with the Medical Research Council framework for complex intervention evaluation, such an approach is required to examine (1) contextual factors influencing implementation, (2) fidelity and variation in delivery, and (3) how service users and professionals experience and respond to the intervention. Objective: This study aimed to evaluate the process of implementing mWorks, specifically focusing on assessing the intervention’s delivery in relation to the context, implementation process, and mechanisms of impact. Methods: This single-case study was bounded by the delivery period of 10 weeks in a primary and specialist mental health service context. During this period, return-to-work professionals (n=2) and service users (n=6) collaborated to initiate mWorks usage. Both qualitative and quantitative methods were used to triangulate multiple data sources. Results: The pandemic and mental health problems posed contextual barriers, particularly during recruitment. However, perceptions of mWorks as a credible and relevant intervention facilitated its implementation. The delivery was performed according to plan, with minimal adaptations. All users adhered to the intervention, and dialogue meetings were highly valued. mWorks was used flexibly according to users’ needs, both during sick leave and at work. The potential impacts included a transformative process for users, fostering acceptance, self-esteem, self-compassion, and a sense of control. It also had the potential to prevent mental ill health, transform negatives into positives, facilitate disclosure of mental health, and support goal setting. The use of quantitative measures for empowerment, engagement, self-efficacy, depression stigma, and quality of life proved feasible and supported the assumptions and direction of results. Conclusions: The recruitment stage of the implementation program encountered significant contextual barriers. However, once the delivery stage began, the implementation of mWorks proved to be feasible. Despite the limited scope of this study, with its small number of participants, the triangulation of data suggests that both users and professionals benefited from mWorks.
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/0f2e619d10828588de6867489083472e" />

Development and Formative Evaluation of a Narrative-Based Serious Game for Pregnancy Education: Mixed Methods Study

Background: Serious games are increasingly used in professional health education and maternal health promotion. However, most pregnancy-related digital interventions target specific behaviors and do not provide a comprehensive, longitudinal simulation of the pregnancy journey that incorporates psychosocial and administrative aspects. Objective: This study aimed to develop and evaluate a narrative-based serious game that simulates the chronological course of pregnancy and to assess its perceived educational usefulness, accessibility, and user acceptance across multiple platforms. Methods: We developed a 9-chapter interactive serious game covering pregnancy recognition, partner communication, public health consultation, mid-pregnancy and late-pregnancy checkups, and home preparation for childbirth. The game was collaboratively created by a pediatrician, 6 medical students, and a student illustrator using a low-cost visual novel engine (TyranoBuilder). It was released in April 2025 on iOS, Android, and Steam. A voluntary, anonymous postgame survey was conducted between April 2025 and January 2026. Descriptive statistics were used to summarize survey responses and platform analytics. This study was approved by the Ethics Committee of Shinshu University Hospital. Results: A total of 65 users completed the postgame questionnaire. Most respondents were aged 10 to 19 years (38/65, 58.5%) and female (55/65, 84.6%). Nearly half of the participants (30/65, 46.2%) completed the game within 1 hour. Gameplay evaluation scores (5-point Likert scale; 3=neutral or appropriate) were balanced: game length (mean 3.37, SD 0.96), difficulty (mean 2.84, SD 0.85), and interactivity (mean 3.31, SD 1.10). Educational outcomes were rated highly (5-point Likert scale; higher=more favorable): reduced anxiety (mean 3.84, SD 0.96), perceived educational usefulness (mean 3.98, SD 1.02), perceived knowledge acquisition (mean 4.06, SD 1.06), story empathy (mean 3.80, SD 1.11), and overall satisfaction (mean 4.05, SD 1.04). Across all platforms, the game achieved 925 cumulative downloads. iOS and Android downloads were predominantly from Japan, whereas Steam downloads were geographically diverse. Of the 21 Steam reviews, 20 (95.2%) were positive. Conclusions: A serious pregnancy education game developed through a low-cost clinician-student collaborative model demonstrated high perceived educational usefulness, balanced gameplay characteristics, and broad user acceptance, including substantial engagement among teenagers and international users. Narrative-based serious games represent an accessible and scalable approach to maternal health education. Further research using more rigorous evaluation designs is warranted to assess long-term educational and behavioral impacts.
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/e2f36ab34de77d0bdb26718b9c29b61f" />

Perspectives on Remote Monitoring via Smartphones and Wearables Among Individuals With Lived Experience or at Risk of Eating Disorders (“This Could Go Very, Very Wrong”): Qualitative Interview Study

Background: Remote measurement technology (RMT) is increasingly used in health research to collect real-world data relevant to clinical states (eg, sleep, activity, and stress). Concerns exist about the impact of remote tracking via personal devices and wearables on individuals with or at risk of eating disorders (EDs) by promoting a focus on exercise, diet, and appearance. There is a lack of research applying RMT to EDs. Objective: This study aimed to explore how smartphone- and wearable-based RMTs influence eating-, exercise-, and weight-related experiences among individuals with a history of or at risk of EDs and to identify perceived benefits, harms, and recommendations for their use in this population. Methods: In total, 14 semistructured interviews were conducted with former participants of Remote Assessment of Disease and Relapse: Major Depressive Disorder, a 2-year digital health study tracking depression outcomes via RMTs. Participants were included in this follow-up if they had disclosed a history of a comorbid ED or were within the at-risk age range (18-30 years) for EDs during Remote Assessment of Disease and Relapse: Major Depressive Disorder and displayed subclinical ED symptoms (Eating Disorder Diagnostic Scale). Interviews explored the impact of app engagement and wearables (Fitbits) on food, activity, and weight-related behaviors and attitudes. Template analysis was adopted to capture themes guided by the focus on ED-relevant domains. Results: In total, 6 themes captured participants’ experiences with RMTs across clinical status and presentation. Participants broadly appreciated the convenience and reflective potential, while some described emotional strain linked to constant self-tracking. Health data impacted participants’ eating and exercise habits through a dynamic process from awareness to cognition to action, fostering healthy routines or obsessive patterns, depending on emotional state, ED presentation, and recovery stage. Self-tracking appeared to mirror illness stage, supporting ED recovery among those with greater distance from illness, but risking reinforcement of compulsive patterns among those with residual or emerging symptoms. Participants’ recommendations for future studies in EDs stressed balancing autonomy with safeguards for vulnerable individuals. Conclusions: These exploratory findings, drawn from individuals with lived ED experience and young people at subclinical risk, suggest that RMT use was shaped by recovery stage and contextual factors, rather than being inherently beneficial or harmful. While findings should not be interpreted as evidence of RMT safety or acceptability in ED cohorts broadly, they raise important questions about ethical RMT design, including the selection of wearables, access to data, and researcher communication with participants.
<img src="https://jmir-production.s3.us-east-2.amazonaws.com/thumbs/b169595d201412fcf0e45228d9bad465" />

GLP-1 Treated Patients with Diabetes Have Raised Risk for Eye Condition

A large study of people with type 2 diabetes suggests that those who started treatment with a glucagon‑like peptide (GLP)‑1 receptor agonist after diagnosis had slightly increased risk of developing a serious eye condition called ischemic optic neuropathy than people with diabetes treated with other medications.

As reported in the Annals of Internal Medicine, the risk for patients given GLP-1 drugs was about twice that of those given a sodium–glucose cotransporter (SGLT)‑2 inhibitor or a dipeptidyl peptidase (DPP)-4 inhibitor although the absolute risk was still low in all groups.

Ischemic optic neuropathy occurs when the optic nerve sustains damage caused by reduced or blocked blood flow, leading to loss of nerve tissue and vision. The main symptom is sudden, usually painless vision loss in one eye, often with missing areas of the visual field that are frequently permanent. It is a rare condition, with 4-10 cases per 100,000 people per year in the U.S., with some factors like age and conditions like type 2 diabetes increasing risk.

In this study, Chintan Dave, PhD, a researcher at Rutgers University, and colleagues included claims data from 161,489 adults aged 18 to 65 years with type 2 diabetes who were newly prescribed a GLP-1 receptor agonist, 122,114 who started a SGLT‑2 inhibitor, and 86,047 who started a DPP‑4 inhibitor.

They excluded anyone who had previously used these drugs or previously had ischemic optic neuropathy. They approximated randomization by balancing more than 80 characteristics across groups. They then collected follow up data for up to 18 months to see who was later diagnosed with ischemic optic neuropathy.

Over 18 months, about nine out of every 10,000 people in the GLP-1 group were diagnosed with ischemic optic neuropathy, compared with about six out of 10,000 in the GLT‑2 inhibitor group and about four out of 10,000 in the DPP‑4 inhibitor group. Essentially there were three to four extra cases of ischemic optic neuropathy per 10,000 patients in the GLP-1 compared with the other groups.

The researchers note that the apparent excess risk was concentrated in older adults, men, and people with more advanced diabetes or eye or cardiovascular conditions.

GLP-1 receptor agonists are now widely used, both in lower doses for treatment of type 2 diabetes and in higher doses to treat obesity.  “Despite the very low absolute risk for ischemic optic neuropathy, the rapidly expanding use of GLP-1 receptor agonists in patients with and without type 2 diabetes increases the clinical and public health importance of any potential association,” conclude the authors.

“Given that type 2 diabetes itself is a risk factor for nonarteritic anterior ischemic optic neuropathy [which constitutes approximately 75% of ischemic optic neuropathy cases] the potential for GLP-1 receptor agonists to further augment this risk has relevant implications for clinical decision making.”

The post GLP-1 Treated Patients with Diabetes Have Raised Risk for Eye Condition appeared first on Inside Precision Medicine.

STAT+: AIDS activists slam Biden R&D deal with Gilead over HIV prevention drug patents

After more than a year of squabbling, a group of AIDS activists obtained an R&D agreement that was at the heart of a settlement between the U.S. government and Gilead Sciences over patents for HIV prevention drugs. But in their view, the deal shows the Biden administration missed a “historic” opportunity to invest in — and expand access to — HIV prevention tools.

As noted previously, the settlement resolved a lawsuit that was filed six years ago by the previous Trump administration after the Centers for Disease Control and Prevention maintained that Gilead infringed on its patent rights. The agency had helped fund academic research that later formed the basis for two Gilead HIV pills, Truvada and Descovy.

The administration had alleged that Gilead ignored the contributions by CDC scientists, exaggerated its own role in developing HIV prevention drugs, and refused to sign a licensing agreement despite “multiple attempts” at reaching a deal after unfairly reaping hundreds of millions of dollars from research funded by taxpayers.

Continue to STAT+ to read the full story…

Analytical Challenges for Antibody-Drug Conjugate (ADC) Manufacturing

Antibody-drug conjugates (ADCs) offer a compelling promise: delivering a cytotoxic payload directly to a tumor cell while sparing the rest of the body from harm. Composed of a tumor-targeting antibody, a cytotoxic agent, and a chemical linker, ADCs combine the selectivity of monoclonal antibodies that bind to tumor-specific antigens with the cytotoxic potency of small-molecule drugs. These therapeutics have the potential to target tumors while reducing systemic toxicity, opening new treatment pathways for many types of cancer. Recent clinical data, including encouraging Phase I findings in platinum-resistant ovarian cancer, validate that potential and fuel the field’s remarkable growth.

Analytical Challenges for Antibody-Drug Conjugate (ADC) Manufacturing cover

Despite the promise of ADCs, their unique structure complicates manufacturing, characterization, and regulatory assessment. Developers must carefully consider the interplay between the antibody, linker, and payload to optimize therapeutic efficacy and safety. The drug-to-antibody ratio (DAR), conjugation site specificity, impurity profile, and linker stability all influence an ADC’s pharmacokinetics, pharmacodynamics, and ultimately its clinical safety profile. Robust analytical methods are therefore critical throughout the development process to ensure both safety and efficacy.

Given the hybrid nature of ADCs, regulatory expectations are still evolving. To navigate this uncertainty, analytical risk management is essential. Moreover, cross-functional collaboration among analytical scientists, process development teams, regulatory experts, and quality assurance professionals is key to ensure that early-stage methods are sufficiently robust and scalable for commercial manufacturing.

Success in this environment requires deep analytical expertise, robust quality-by-design frameworks, and development partners who understand the full arc from early-stage linker-payload synthesis through GMP-compliant manufacture. It requires the capability to handle highly potent compounds safely, to purify structurally complex intermediates at scale, and to translate rigorous quality control into processes that are commercially viable.

This collection of articles and expert perspectives explores the critical challenges shaping ADC development today, from analytical strategy and impurity control to linker technology innovations and evolving regulatory standards. It also examines the collaborative expertise needed to bring these transformative therapies to patients.

The post Analytical Challenges for Antibody-Drug Conjugate (ADC) Manufacturing appeared first on GEN – Genetic Engineering and Biotechnology News.

New Lung Model Reveals RSV Immune Response, Points to Better Treatments

Researchers at University College London (UCL) Great Ormond Street Hospital for Children (GOSH) have built a new lab model of infant lungs to show why respiratory syncytial virus (RSV) makes infants so much sicker than adults, and to allow them to test new treatments. The miniature model of a baby’s airways was created using pediatric airway cells, blood vessel cells, and neutrophils (a type of white blood cell that acts as the immune system’s primary response to infection).

Studies using the new model suggest that future therapies for RSV should target both the virus and its immune response to ensure babies get the best possible outcomes. Research lead Claire Smith, PhD, at UCL Great Ormond Street Institute of Child Health, said, “This model allows us to watch early immune responses unfold and study them in a human setting that reflects the infant airway. That’s something animal models often struggle to capture, especially when it comes to age-specific effects.”

Senior and corresponding author Smith and colleagues reported on their findings in Nature Communications in a paper titled “Neutrophil myeloperoxidase as a functional biomarker for RSV severity: implications for in vitro therapeutic screening.” In their report they concluded, “These findings identify neutrophil–epithelial interactions as a useful target for intervention and support the use of physiologically relevant human models to accelerate the development of therapies that limit immunopathology while preserving antiviral defense.”

RSV is the biggest cause of severe respiratory tract infections in infants and young children, resulting in over three million hospital admissions worldwide every year. RSV infection causes wheezing and breathing difficulties, and in the worst cases infants end up in intensive care. Despite this, treatment options for severe RSV disease remain extremely limited. “The lack of accessible, effective therapies highlights the need for continued advancements in RSV treatment and prevention,” the authors wrote.

During RSV infection neutrophils are rapidly mobilized to the lungs and play a key role in virus-targeting host defenses, the investigators continued. However, excessive neutrophil infiltration and activation can also contribute to airway inflammation, epithelial damage, and disease severity. “Understanding neutrophil behavior and activation during RSV infection, including during their migration across the airway epithelial barrier, is crucial for developing therapeutic strategies to mitigate pathological inflammation without compromising the antiviral response,” they suggested.

For their reported study the team aimed to create an in vitro model that recapitulates key clinical outcomes of infants with RSV bronchiolitis. To do this they established an air-liquid interface (ALI) system that incorporated pediatric airway epithelial cells, endothelial cells, and neutrophils from adults, to mirror the blood-airway barrier. “Differentiated airway epithelial cells (AECs) cultured at the air–liquid interface (ALI) provide a physiologically relevant platform to study neutrophil migration and the effect of antiviral treatments on this process,” they note. To compare with an adult response to RSV, the research team also made a model of an adult’s airways.

When the models were infected with RSV, the team found that the pediatric airway cells attracted far more white blood cells than did the adult airway cells. This influx can block an infant’s small airways and make it harder for them to breathe.

Neutrophils normally circulate in the blood but enter lung tissue in response to infection. In the baby airway model, researchers found that the neutrophils that entered the lung tissue were more activated and triggered a stronger inflammatory reaction than in the adult model. This effect was dependent on the immune cells physically moving through the infected tissue, not just responding to chemical signals released by it, making this type of model essential for study.

The results suggest that it is the infant airway itself, not just the virus, that ramps up the immune response and causes damage to the lungs. First author Machaela Palor, PhD, at UCL Great Ormond Street Institute of Child Health, said: “These findings help explain why RSV is often much more severe in infants than in adults. The pediatric airway actively shapes how immune cells behave during the infection.”

The researchers tested two antiviral drugs (remdesivir and RSV604). they found that both stopped the virus from multiplying, but only RSV604 also calmed the overactive immune response, reducing levels of a key inflammatory protein myeloperoxidase (MPO) released by white blood cells—high levels of which are linked to more severe RSV disease in babies. Remdesivir had no effect on this, suggesting that not all antivirals are equal when it comes to protecting the infant airway from immune-driven damage. “While both drugs reduced viral load, only RSV604 attenuated MPO expression,” the team stated.

The findings suggest that treating severe RSV in babies may require more than just stopping the virus—it may also be important to calm an overactive immune response. “This model suggests that MPO could be useful as a readout of therapeutic efficacy,” the team stated. “Targeting neutrophil-driven inflammatory pathways may be critical for reducing pathology in infant RSV infection.”

The researchers hope their findings and the new approach to research on RSV will accelerate the development of treatments better tailored to infants. “Our model gives us a way to assess both sides of the problem at once,” Smith said. “We can not only ask whether the drug stops the virus but also whether it helps control immune response in the infant airway. “This work reinforces the idea that age matters in respiratory infection. Understanding how infant airways shape immune responses will be key to designing safer and more effective RSV treatments.”

The post New Lung Model Reveals RSV Immune Response, Points to Better Treatments appeared first on GEN – Genetic Engineering and Biotechnology News.

Targeting Microglia Could Help Extend the Recovery Window After Stroke

Stroke care has advanced most clearly in the acute phase: rapid reperfusion, prevention of edema, secondary prevention, and early rehabilitation. Yet for many patients, the most difficult clinical reality begins after stabilization. Neurological recovery often improves over weeks to months, then plateaus. Once that spontaneous recovery window closes, residual motor, language, or cognitive deficits may become permanent.

A study published in Nature now identifies a molecular mechanism that may help explain why this window narrows. Researchers led by Jun Tsuyama and Takashi Shichita, PhD, at the Institute of Science Tokyo found that microglia, the brain’s resident immune cells, can remain in the post-stroke brain after losing their reparative function. The team identified ZFP384 as a transcriptional regulator that suppresses the microglial repair program and showed that blocking it with an antisense oligonucleotide improved long-term recovery in mouse models of ischemic stroke.

A repair program that fades too soon

Microglia are often discussed in the context of neuroinflammation, but their role after stroke is not uniformly harmful. In the acute phase, activated myeloid cells contribute to inflammatory injury. During recovery, however, microglia can shift toward a reparative state, producing neurotrophic and tissue-supportive factors that contribute to remyelination, synaptic remodeling, and functional improvement.

One of the key markers in this study was insulin-like growth factor 1, or IGF1, a neurotrophic factor produced by reparative microglia. IGF1 has known roles in synaptogenesis, oligodendrocyte function, and myelin repair. The researchers used IGF1 expression to track microglia involved in the recovery phase after ischemic stroke.

“We aimed to identify the molecular mechanism responsible for diminishing microglial reparative functions,” Tsuyama said in the press release.

The central observation was clinically relevant: reparative microglia did not simply disappear. Instead, lineage-tracing experiments showed that cells which had once expressed repair-associated genes persisted in the peri-infarct region but later lost that gene-expression program. In mice, recovery-associated gene expression rose after stroke and then declined toward baseline by around day 28. The cells remained, but their reparative identity faded.

ZFP384 as a brake on microglial repair

To identify what shuts down this repair state, the researchers combined RNA sequencing, single-cell RNA sequencing, ATAC-seq, and transcription-factor analysis. A protein known as ZFP384 emerged as a candidate regulator whose expression increased as the microglial repair program declined.

Functionally, ZFP384 acted as a brake. Overexpression of Zfp384 reduced Igf1 expression in microglial cells, while genetic deletion of Zfp384 in microglia sustained recovery-associated gene expression after stroke. Mice lacking Zfp384 specifically in microglia showed better long-term neurological outcomes on behavioral tests, without significant differences in infarct volume, cerebral blood flow, or survival.

That distinction is important. The intervention did not appear to reduce the initial ischemic injury. Instead, it improved the recovery phase, suggesting a therapeutic concept distinct from acute neuroprotection.

Mechanistically, the study links ZFP384 to disruption of YY1-mediated chromatin interactions. YY1 helped maintain enhancer–promoter contacts required for recovery-associated gene expression, including at the Igf1 locus. As ZFP384 increased, it displaced this repair-permissive chromatin organization, shifting microglia toward a dysfunctional state.

The authors describe this as a strategy to “prevent the loss of reparative immunity,” preserving beneficial immune-cell functions rather than broadly suppressing inflammation.

Antisense therapy improved recovery in mice

The translational component of the study used antisense oligonucleotides (ASO) designed to reduce Zfp384 expression. After intracerebroventricular administration, the ASO was taken up by microglia and reduced Zfp384 mRNA expression.

When ASO-Zfp384 was administered on days 8 and 22 after stroke onset, mice showed improved neurological recovery compared with controls. Notably, benefit was also observed when treatment began on day 29, suggesting that the approach may influence the chronic recovery phase in this mouse model rather than only early repair.

The biological readouts supported the behavioral findings. ASO-Zfp384 sustained microglial recovery-associated gene expression, increased IGF1-positive microglia in peri-infarct tissue, and promoted broader neural repair signatures in oligodendrocyte precursor cells, excitatory neurons, and astrocytes. Treated mice showed improved myelination, enhanced white matter conduction, and increased synaptic markers including synaptophysin and PSD95.

Neutralizing IGF1 or SPP1 reduced the recovery benefit, strengthening the conclusion that microglial neurotrophic factors were functionally involved.

Human tissue supports the pathway

The researchers also examined human post-mortem brain tissue from patients who had experienced ischemic stroke. In peri-infarct regions, IGF1-positive IBA1-positive cells were more abundant early after stroke and declined later. ZNF384, the human orthologue of mouse ZFP384, showed the opposite pattern, increasing later in the recovery period.

The inverse relationship between IGF1 and ZNF384 supports the relevance of the pathway in human stroke biology, although it remains correlative. The human samples do not show that ZNF384 inhibition would improve patient outcomes, but they indicate that the same molecular pattern observed in mice may also occur in the human post-stroke brain.

Implications for rehabilitation medicine

The findings point to a therapeutic space that remains underdeveloped: enhancing recovery after the acute phase. Rehabilitation depends on plasticity, remyelination, and circuit remodeling, but current pharmacological options to extend or potentiate this biology are limited.

A therapy that sustains reparative microglia could, in principle, complement rehabilitation by keeping the peri-infarct environment more permissive for repair. That would represent a different clinical goal from thrombolysis, thrombectomy, or anti-inflammatory intervention. Rather than rescuing threatened tissue in the first hours, the aim would be to improve the quality and duration of recovery over subsequent weeks.

The study remains preclinical. Delivery route, dose, safety, timing, durability, and patient selection all require further work. The mouse model cannot capture the full heterogeneity of human stroke, including lesion location, age, comorbidities, vascular risk, rehabilitation intensity, and medication use. Sustaining immune-mediated repair also needs careful safety evaluation, because prolonged activation of tissue-resident immune cells could have context-dependent risks.

Still, the concept is compelling. Microglia are not merely inflammatory cells to inhibit; they can be repair partners whose beneficial state may be actively preserved. If ZFP384-targeted approaches prove safe and effective in larger models, they could open a new class of post-stroke recovery therapies focused on extending the brain’s own repair window.

As Tsuyama put it, “sustaining the brain’s endogenous repair program” may create opportunities to reduce permanent neurological symptoms during rehabilitation.

The post Targeting Microglia Could Help Extend the Recovery Window After Stroke appeared first on Inside Precision Medicine.

Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury

A stem cell therapy designed to restore function by repairing damaged nerve pathways is moving closer to clinical testing for people with chronic spinal cord injury (SCI), according to research presented at the International Society for Stem Cell Research (ISSCR) 2026 Annual Meeting.

The work, presented by Hideyuki Okano, MD, PhD, of Keio University in Japan, builds on more than a decade of research into regenerative therapies for SCI. Okano’s team pioneered the development of induced pluripotent stem cell (iPSC)-derived neural stem/progenitor cells and launched the world’s first clinical study of the approach in patients with subacute SCI.

In March 2025, the researchers announced one-year follow-up results from that four-patient first-in-human study, reporting no serious treatment-related adverse events and neurological improvements in two participants, including one who regained the ability to stand independently and begin gait training. Although those findings have not yet been published in a peer-reviewed journal, they provided the safety foundation for expanding the program to chronic SCI.

The physician-initiated trial, expected to begin recruiting patients in 2027, will enroll individuals with chronic, incomplete SCI who retain inactive but demyelinated nerve fibers. Unlike patients treated soon after injury, these individuals often have surviving nerve pathways that have lost the myelin insulation needed for efficient signal transmission.

“We have already successfully completed a world-first, first-in-human clinical study targeting patients in the subacute phase, which demonstrated a promising safety profile,” Okano said. “Our shift to the chronic phase is the next logical milestone, built upon that solid foundation. Since the cellular environment changes over time, we are evolving our strategy from just establishing safety to actively overcoming the stubborn, long-standing barriers of chronic paralysis.”

The biology of chronic injury also required a different cell therapy. While the earlier program used neural stem/progenitor cells capable of generating neurons, the new approach uses clinical-grade gliogenic neural stem/progenitor cells (gNS/PCs), which preferentially develop into astrocytes and oligodendrocytes—the support cells responsible for maintaining neurons and restoring myelin around surviving nerve fibers. Rather than attempting to regenerate entirely new neural circuits, the therapy aims to repair and restore the function of existing ones. Earlier preclinical studies demonstrated that these gliogenic cells promoted remyelination, preserved host axons, and improved locomotor recovery in animal models, providing the rationale for advancing the therapy toward human testing.

The latest preclinical findings further support that strategy. The researchers reported that the clinical-grade gNS/PCs efficiently differentiated into neurons, astrocytes, and oligodendrocytes in laboratory studies. Following transplantation into animal models of chronic SCI, the cells promoted behavioral recovery without evidence of tumor-like tissue formation while remodeling the injured spinal cord microenvironment.

The post Stem Cell Therapy Advances Toward Clinical Trial for Chronic Spinal Cord Injury appeared first on Inside Precision Medicine.