Articles tagged with Gene Therapy
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 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.
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.
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.
A new approach enables hospital pharmacists to rapidly create bespoke medicine cartridges for rare disease patients, boosting access to personalized treatment. The NANOSPRESSO platform could open up treatments for underfunded and underserved rare conditions worldwide.
A phase 3 clinical trial shows that genetically engineered skin grafts treat severe dystrophic epidermolysis bullosa, reducing pain and healing time. The treatment is part of a larger effort to improve EB patients' treatment options.
Researchers have found a promising new method for gene therapy by bringing dormant genes closer to enhancer switches on the DNA. This 'delete-to-recruit' strategy has potential for treating genetic diseases such as sickle cell disease and beta-thalassemia, offering an alternative to expensive current treatments.
The National Urea Cycle Disorders Foundation is establishing a multistakeholder Partner Network to guide health care decisions and build a sustainable infrastructure for UCD research. The project aims to empower the broad UCD community to work together effectively, identify research needs, and create a roadmap for future studies.
Researchers at The University of Osaka have discovered a molecular mechanism behind genome ejection from adeno-associated virus (AAV) vectors. The study reveals that the N-terminal region of the VP1 protein undergoes structural changes upon heating, facilitating genome release.
Researchers at KAIST have developed a groundbreaking technology capable of selectively acetylating specific RNA molecules within the human body using the CRISPR-Cas13 system. This breakthrough enables precise, programmable control of RNA function and is expected to open new avenues in RNA-based therapeutic development.
Researchers have discovered a novel cell-clearance pathway linked to diseases such as Chediak-Higashi Syndrome, which affects immune system function. The study used CRISPR/Cas9 gene-editing technology and live imaging to characterize this pathway and identify key genes involved.
Researchers have developed a gene therapy that targets the root cause of Alzheimer's disease, influencing brain cell behavior to preserve cognitive function. Delivering the treatment at the symptomatic stage preserved hippocampal-dependent memory and altered gene expression in mice, suggesting potential to restore brain health.
Stanford researchers have developed a machine learning approach to design proteins that can target specific genomic sites without triggering immune responses. By combining three independent algorithms, the team created zinc finger DNA-binding domains with improved functionality and lowered immunogenicity.
A new study identifies CSMD1 as a novel gene associated with generalized epilepsies, including developmental and epileptic encephalopathy. The research found that CSMD1 variants are more damaging and exhibit lower minor allele frequencies in DEE cases compared to IGE cases.
A team of scientists from SR-Tiget has identified a unique window shortly after birth to deliver lentiviral vectors directly into the bloodstream, enabling gene transfer and long-term engraftment. This approach shows promise for treating some genetic blood disorders without stem cell transplantation or chemotherapy.
Researchers at the University of Pennsylvania developed novel promoters that drive specific gene expression in rod and cone photoreceptors, outperforming most currently used promoters. These tools address the challenge of treating advanced stages of inherited retinal diseases, potentially restoring vision.
Researchers developed a gene therapy that can target the airway and lungs using a nasal spray, outperforming previous versions in preclinical models. The innovative tool, AAV.CPP.16, showed promise for treating respiratory diseases like pulmonary fibrosis and viral infections.
Scientists have uncovered a previously unknown mechanism explaining how neurons survive botulinum neurotoxin type A exposure. The research found that specific tRNA fragments interact with key proteins and RNA molecules involved in regulating ferroptosis, supporting neuronal survival by blocking cell death pathways.
A child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy, showcasing the power of tailored gene editing to treat patients. The infant is now growing well and thriving after receiving three doses of the therapy with no serious side effects.
Lurie Children's is now a Qualified Treatment Center (QTC) for ZEVASKYN, the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB). This therapy promises to provide long-term healing of wounds, reduction in pain and reduced risk of infection.
Researchers from The University of Osaka developed a new technique using mass photometry to detect and quantify components of rAAV particles. This method can distinguish between full and empty particles, streamlining gene therapy manufacturing and improving clinical effectiveness.
Researchers found that supplements of B vitamins B6, B9, and B12, as well as choline, slowed down damage to the optic nerve in glaucoma. This was observed in experiments on mice and rats with glaucoma, where eye pressure was left untreated.
Researchers have successfully tested a CRISPR/Cas9 gene-editing technique to enhance the immune system's fight against advanced gastrointestinal (GI) cancers. The treatment showed encouraging signs of safety and potential effectiveness in patients with stage IV colorectal cancer, halting tumor growth and even achieving complete responses.
A new gene therapy has successfully restored immune function in nine children with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder. The treatment has eliminated symptoms and reduced severe infections, offering a life-changing benefit to these patients.
Researchers at Karolinska Institutet have developed a technique to deliver gene editors and protein therapeutics to cells using engineered extracellular vesicles. The method shows promising results in animal studies, highlighting the potential for treating genetic diseases and neurological disorders.