Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
A new study by McGill University researchers offers a potential solution for infant hydrocephalus, a life-threatening condition that affects 1 in 1,000 newborns. RNA therapy has been shown to prevent the condition in mice models, with a promising impact on treating genetically caused hydrocephalus.
The ISSCR is hosting a summit on access and affordability in cell and gene therapies to explore pricing, manufacturing, regulation, and reimbursement strategies. Experts will examine key barriers and emerging solutions across the access landscape.
Researchers at University of California San Diego discover gene therapy restoring connexin-43 improves heart function and extends survival in several forms of arrhythmogenic cardiomyopathy. The approach may have broader therapeutic potential across multiple genetic forms of the disease, addressing a critical unmet need.
The collaboration aims to deliver life-saving treatments at a fraction of their current market cost, using Caring Cross's decentralized manufacturing model. Clinical data demonstrated robust increases in fetal hemoglobin with sustained mitigation of SCD disease manifestations.
Researchers at Rice University are exploring biological systems-inspired delivery vehicles to target specific tissues in living organisms, aiming to improve the efficiency of gene-based therapies. The project focuses on optimizing combinations of surface molecules to enable precise and efficient delivery of large DNA payloads.
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
The CRISPR-Cas3 system has been shown to induce reliable and extensive deletions of the TTR gene in mouse models of ATTR, reducing serum TTR levels by up to 80%. This technology holds promise for treating not onlyATTR but also other incurable inherited diseases.
The Rice Brain Institute has awarded seed grants to four collaborative projects that unite Rice faculty with clinicians and scientists across the Texas Medical Center. The grants support innovative research in brain science, mental health, and neurological disease.
A University of California San Diego team is developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia. The $7.4 million grant will support safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients.
Researchers at Drexel University and UMass Chan Medical School have developed a gene therapy approach that silences and replaces disease-causing genes in mice with hereditary spastic paraplegia. The treatment prevents nerve breakdown and symptoms, offering a potential cure for the rare disease. Studies suggest that the therapy could be...
Waskyra, an ex vivo gene therapy, offers new hope for patients with Wiskott-Aldrich syndrome by reducing severe bleeding events and serious infections. The FDA approval confirms Fondazione Telethon's excellence in rare genetic disease research.
A study assessing the commercial roll-out of gene therapies for sickle cell disease and beta thalassemia reveals operational differences between two treatments. The median time to complete treatment was around 9-10 months, with most patients requiring only one cell collection procedure.
Preliminary results from trials of gene therapy exa-cel suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12. The therapy's potential to prevent irreversible complications makes it potentially more beneficial in children than adults.
Researchers at Rice University have developed a way to make serum markers more sensitive by editing them inside the bloodstream, enabling clearer observation of gene-expression changes in the brain. This approach could lead to more precise diagnostic capabilities using simple blood tests.
The review highlights the progress in gene-editing nucleases and delivery vectors for in vivo gene therapy, enabling targeted correction of genetic errors. Key examples include FDA-approved products targeting monogenic disorders and cancers, showcasing the therapeutic promise of combining genome editors with advanced delivery platforms.
Researchers have discovered a new gene, ALOX15, that protects against sepsis-induced lung injury in mouse models. The findings suggest that mild lung thrombosis may actually reduce the severity of lung damage from sepsis, offering a potential novel therapeutic strategy.
Researchers have shown that disabling the NRF2 gene with CRISPR technology can restore drug sensitivity and slow tumor growth in lung cancer. The approach, which targets a master switch for resistance, has potential across multiple tumor types.
A nonsurgical approach has been demonstrated to quiet a specific brain circuit in an animal model by delivering engineered gene therapy only to the targeted region. The method uses low-intensity focused ultrasound to open the blood-brain barrier, allowing precise control over brain activity without impacting off-target areas.
Researchers at Uppsala University have identified a new genetic technique that enables targeting of tumour cells with elevated levels of proteins driving medulloblastoma. The technique, which works like a Trojan horse, introduces an enzyme linked to SOX9 sequence, killing tumour cells.
Researchers at Weill Cornell Medicine have developed a powerful new gene-switch tool called Cyclone, which allows scientists to turn on or off target genes with precision. The tool uses a non-toxic molecule acyclovir to suppress gene activity, and has the potential to be adopted throughout biomedical research and gene therapies.
A naturally occurring gene called Cyclin A2, normally silenced in humans, can make new functioning heart cells and aid in the heart's repair. The breakthrough discovery could lead to new techniques for repairing damaged hearts as an alternative to transplants or implanted cardiac devices.
International experts have established guidelines for gene therapy in hereditary hearing loss, emphasizing patient-centered care and respect for diversity. The guidelines outline frameworks for clinical trials, including patient selection criteria, surgical procedures, and safety evaluations.
A new gene therapy has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare genetic immune disorder. The treatment involves delivering a healthy copy of the ADA gene to patients' blood stem cells, allowing them to produce healthy immune cells capable of fighting infections.
A landmark study published in the New England Journal of Medicine reports the long-term safety and efficacy of gene therapy for children with ADA-SCID, a rare immune disorder. The treatment resulted in a 100% survival rate and over 95% cure rate, with patients able to respond to routine childhood vaccinations.
Researchers at MIT have developed a new system that allows for precise control over the expression of synthetic genes in cells. The DIAL system uses a promoter editing mechanism to establish desired protein levels, which can be edited after delivery. This technology has the potential to improve gene therapy and cell reprogramming appli...
Researchers developed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders in mice, including intellectual disability, epilepsy, and risk-taking behaviors. The therapy successfully delivered a working copy of the SYNGAP1 gene into brain cells using an adeno-associated virus, offering hope for treatment in humans.
Commercial health plans in the US impose different coverage requirements for FDA-approved cell and gene therapies, often aligning with pivotal trial criteria. These restrictions suggest a need for stronger evidence to support plan coverage.
A new biotechnical vector, VIBV, combines viral mimicry with synthetic nanotechnology to deliver targeted RNA therapies for cancer treatment. The vector uses a spindle-shaped nanostructure and polyethylene glycolylated liposomal coat to evade immunity and extend circulation.
Maria Jasin, a renowned developmental biologist, has been awarded the 2025 Pearl Meister Greengard Prize for her groundbreaking work on DNA repair. Her research has transformed our understanding of cancers linked to inherited gene mutations, and has led to significant advances in cancer treatment.
Researchers from Mass General Brigham developed a bespoke CRISPR-Cas9 gene-editing enzyme to correct the genetic error causing multisystemic smooth muscle dysfunction syndrome, a rare condition associated with stroke and death in childhood. The therapy extended survival four-fold in mouse models of MSMDS.
A study published in the Journal of Hepatology reveals that only 15-20% of neonatal liver cells are responsible for generating over 90% of the adult liver mass. This finding has major implications for pediatric gene therapy, allowing scientists to achieve more effective and durable correction of inherited liver diseases.
Treatment with exagamglogene autotemcel (exa-cel) leads to clinically meaningful improvements in overall quality of life for patients with severe sickle cell disease and transfusion-dependent beta thalassemia. Patients experience substantial improvements in physical, social, functional, and emotional well-being, with sustained benefits...
The EASYGEN consortium will develop a fully automated platform for manufacturing personalized cell therapies within days, accelerating patient access and reducing costs. EBMT is participating in the study examining current CAR-T treatment processes and contributing to patient education and advocacy efforts.
Researchers at the University of Waterloo have developed a novel method using modified M13 bacteria to deliver targeted gene therapies for genetic disorders. This approach shows promise as a cost-effective alternative to current methods, which can be expensive and trigger toxic side effects.
Researchers have identified the genetic cause of feline atherosclerosis, an arterial disease in cats. The discovery may help prevent the incidence of the disease in cats and possibly open new courses for human therapies.
Macromolecular gene delivery systems are advancing non-viral therapeutics by overcoming challenges like lower transfection efficiency and stability issues. Innovations in polymer design, functionalization, and targeting mechanisms are paving the way for clinically viable non-viral treatments.
Researchers used human stem cell-derived kidney organoids to test the safety of gene editing delivered by AAV, a common tool in clinical trials. The study found that AAV2 caused significant harm to kidney cells through the NFκB pathway, but an existing drug was able to prevent this damage without interfering with gene delivery.
A new study sheds light on how viruses pack their genetic material with high selectivity, achieved with over 99% accuracy. Understanding this process could help create lab-made versions of capsids for gene therapy and antiviral development.
Researchers developed a one-time gene editing treatment that restored hearing and balance in adult mice with DFNA41, a genetic form of progressive deafness. The therapy successfully disabled the harmful mutation while preserving the normal gene, leading to long-term hearing and balance restoration.
Researchers at North Carolina State University have developed a controlled evolution technique that dramatically increases plasmid DNA (pDNA) production in E. coli bacteria. This breakthrough could significantly reduce the cost of gene therapies and expedite research, making pDNA resources more accessible.
A research team developed a new method to precisely edit DNA by combining genetic engineering with artificial intelligence. The technique enables accurate modeling of human diseases and lays the groundwork for next-generation gene therapies.
Researchers have developed a groundbreaking gene therapy approach for treating neovascular age-related macular degeneration, targeting VEGF-A and ANG-2 to suppress abnormal blood vessel growth. The treatment has shown significant clinical benefits in animal models and patients with minimal side effects.
A new gene therapy strategy has shown multi-year protection from HIV/AIDS infection in newborns via broadly neutralizing antibodies. The treatment, administered once at birth, could prevent HIV-1 transmission during breastfeeding, especially in areas where access to antiretroviral medications is limited.
A new study shows that delivering a single injection of gene therapy at birth may offer years-long protection against HIV. The treatment uses an adeno-associated virus to deliver instructions to muscle cells, which produce broadly neutralizing antibodies capable of neutralizing multiple strains of HIV.
A new gene therapy intervention administered at birth provides multi-year protection from HIV infection, useful in areas lacking access to antiretroviral drugs. The treatment uses a common gene therapy shuttle to produce an HIV-specific broadly neutralizing antibody, showing no side effects and long-lasting protection.
Genethon is launching a Phase 3 clinical trial in Europe for its low-dose microdystrophin gene therapy GNT0004, targeting boys aged 6 to 10 with retained walking ability. The trial aims to demonstrate efficacy and tolerance of the treatment.
Researchers at UC Davis Health developed a promising gene therapy that could treat Rett syndrome by reactivating healthy but silent genes responsible for this rare disorder. The therapy showed impressive results in female mouse models of Rett syndrome, with treated mice living longer and showing better movement and cognition.
A new study reveals B cell-derived ELL2 as a promising biomarker for diagnosing and predicting the prognosis of sepsis. The research identified three subtypes with significantly different prognoses, which were consistently reproduced across all cohorts.
Researchers at the University of Pennsylvania designed a new recipe for mRNA vaccines by adding phenol groups, which reduce inflammation and improve vaccine effectiveness. The modified lipids improved vaccine performance in various diseases, including COVID-19, cancer, and genetic diseases, with enhanced efficacy and reduced side effects.
The FDA has granted orphan drug designation to a new gene therapy for Amyotrophic Lateral Sclerosis (ALS), a devastating disease with no current effective treatment. The therapy uses a viral vector expressing the secreted isoform of Klotho protein, showing promising results in preclinical studies.
Gene therapy relies on efficient and safe delivery of therapeutic genes to target cells. Macromolecular carriers, including synthetic and natural polymers, offer biocompatibility, controlled release, and targeted delivery. These systems have shown promise in treating genetic disorders and complex diseases like cancer.
Scientists have identified a new gateway into human cells that could make gene therapies safer and more effective. The discovery of AAVR2 receptor allows for lower doses of virus to be used in treatment, reducing side effects and costs while improving patient outcomes.
The Center will develop personalized CRISPR on-demand treatments for children with severe inborn errors of immunity and metabolic disease. The initiative aims to make CRISPR cures more affordable and accessible, building on recent clinical success in treating ultra-rare genetic diseases.
Scientists are working on a genetic cure for the world's most common inherited heart disease, hypertrophic cardiomyopathy (HCM), with a new gene editing tool. The team aims to correct mutations that cause the disease, which affects 14 million people worldwide.
Researchers at Case Western Reserve University have received an NSF CAREER Award to develop synthetic DNA nanoparticles with potential applications in gene therapy. The program aims to study how these particles behave inside cells and potentially design therapeutics for genetic diseases.
A new cytokine delivery platform reprograms the tumor microenvironment to enhance CAR-T cell function in preclinical brain cancer models. The strategy leads to a broader immune response that inhibits tumor growth and extends host survival, even in mice with only a fraction of cells expressing the CAR-targeted antigen.
A new study conducted at Karolinska Institutet reports that gene therapy improved hearing in all ten patients with congenital deafness or severe hearing impairment. The treatment was well-tolerated and showed remarkable results, with the majority of patients recovering some hearing after just one month.
Researchers aim to replenish the heart's energy reserves by increasing mitochondrial biogenesis, potentially leading to improved recovery rates and longer lives after a heart attack. The project combines synthetic biology tools with animal models and human heart tissue testing.
Gene therapy has been shown to significantly improve blood flow in the brains of patients with sickle cell disease, decreasing the risk of stroke. The treatment has a more substantial and long-lasting protective effect than other treatments like hydroxyurea or blood transfusions.
A new gene therapy delivery device called NANOSPRESSO could revolutionize how hospitals treat rare diseases by allowing them to create personalized nanomedicines in-house. This democratized approach to precision medicine could boost access to low-cost bespoke gene and RNA therapies, especially in low-resource settings.