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 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.
A new CAR-T therapy, HSP-CAR30, has achieved positive results in a high proportion of patients with refractory CD30+ lymphoma. The treatment promotes the expansion of memory T cells, leading to durable responses and improved clinical outcomes.
Researchers at MIT developed a control circuit that can precisely regulate gene expression levels, improving the efficacy and safety of gene therapy treatments. The 'COMMAND' circuit uses microRNA to suppress gene expression, allowing for tighter control over treatment outcomes.
Researchers develop nanoparticle-based therapy combining hydroxyl-enriched fullerenol and mTOR inhibitors to disrupt cancer cells' organelle communication system. The approach triggers a synergistic "nanomaterial + metabolic modulation" anticancer strategy, establishing a new hope for treating aggressive cancers.
The treatment demonstrated early signals of efficacy, with 65.7% of patients experiencing lasting stable disease, and was generally well-tolerated, with most adverse events being mild and manageable.
A Phase II trial found durable antitumor activity in patients with BRCA1/2 mutations treated with olaparib and pembrolizumab, with 8.3% complete response rate
A new study reports on five patients with Canavan disease who have a novel variant identified through targeted long-read sequencing, revealing an SVA_E retrotransposable element that disrupts gene function. The findings enhance genetic diagnostics and enable improved guidance for families.
A recent study published in the New England Journal of Medicine found that gene therapy can significantly reduce the risk of severe motor and cognitive impairment in children with metachromatic leukodystrophy (MLD). The therapy, administered early, preserves motor function and cognitive abilities in most patients.
A UC Riverside-led study found that adult stem cells rely on histone chaperones to maintain their regenerative capacity. The researchers discovered that disrupting these proteins can lead to specific changes in stem cell identity, potentially guiding them into desired cell types.
Novel biomarkers like miRNA-34a link anthracyclines to cardiotoxicity, while stem cell therapy and nanotechnology offer potential for prevention and treatment. Traditional strategies have limitations, but new approaches hold hope for improved patient outcomes.
A team of researchers has developed a gene-therapy strategy to treat arrhythmogenic right ventricular cardiomyopathy type 5 (ARVC5), a rare and deadly hereditary disease. The treatment, using adeno-associated viruses, improved cardiac function and prolonged survival in mice with ARVC5-like disease.
A recent trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, with the immune system emerging as a key barrier to its success. Researchers identify early intervention at birth and personalized immune screening as potential solutions to overcome this hurdle.
Researchers found that NSD2 helps maintain MM cell identity by reorganizing DNA and influencing gene activity. This discovery could shape future treatment approaches for patients with t(4;14) myeloma.
A multidisciplinary team has generated an atlas to optimize gene therapy delivery, providing researchers with insights into the most effective viral vectors for specific tissues. The study identifies AAV4 as a promising vector for vascular and pancreatic applications, offering new possibilities for treating conditions like diabetes.
A recent study demonstrates gene therapy's potential to restore motor capacity in an ultra-rare disease, Megalencephalic Leukoencephalopathy with Subcortical Cysts (MLC), even after symptom onset. The treatment restored normal protein activity, normalizing physiological brain alterations and reversing motor impairments in treated mice.
Researchers at MD Anderson Cancer Center made several key discoveries, including the spatial organization of cancer-associated fibroblasts across various cancers and a study on treatment resistance in SMARCA4-mutant lung cancer. These findings highlight the importance of investigating cell populations in their spatial context to better...
Researchers at the NIH developed eye drops containing a short peptide derived from PEDF, which slowed photoreceptor degeneration and vision loss in animal models of retinitis pigmentosa and dry age-related macular degeneration. The treatment preserved up to 75% of photoreceptors and showed no negative side effects.
Scientists have developed a new gene replacement therapy in mice that alleviated symptoms without side effects, offering long-term recovery for patients. The therapy targets the SCN1A gene, which plays a crucial role in regulating brain activity, and shows promising results in treating Dravet syndrome.
Researchers at Rice University have gained insights into ADAR1's molecular mechanisms, which could lead to improved treatments for cancer and autoimmune diseases. The study found that ADAR1's editing activity depends on RNA sequence, duplex length, and mismatches near the editing site.
Researchers have developed a new gene therapy protocol using the SIN-EFS-IL2RG.co vector, which demonstrates safety and efficacy in preclinical studies. The treatment restores immune functions and lacks oncogenicity, paving the way for further clinical trials in X-SCID patients.
Researchers have discovered new CRISPR-Cas systems with improved efficiency and specificity, including one from dairy cow bacteria that can target specific gene sequences. The new systems have potential applications in human health, biotechnology and environmental fields.
Researchers are conducting a first-in-human clinical trial to test a modified herpes virus that targets spinal cord nerve cells to treat neurogenic bladder. The therapy, EG110A, aims to block sensory nerve signals causing involuntary bladder contractions and incontinence.
Researchers discovered a molecular switch, FLI-1, essential for blood stem cells to enter an activated state. Transiently producing FLI-1 in quiescent adult mobilized bone marrow stem cells activates them, improving their ability to expand and restore the blood cell supply in a new host.
The new AI model leverages hypergraphs to quickly and accurately identify therapeutic gene targets for diseases. HIT outperformed existing models in all tested metrics, demonstrating its accuracy in classifying therapeutic gene targets with great precision.
A new study reveals that radiotherapy has opposite effects on glioblastoma multiforme (GBM) and low-grade gliomas (LGG), with GBM patients living longer after treatment. The study highlights the need for personalized treatment approaches based on genetic and molecular characteristics to improve survival outcomes.
A gene therapy treatment has demonstrated significant promise in treating maple syrup urine disease (MSUD), a rare genetic disorder. The therapy prevented death and normalized growth in animal models, including a calf with the condition, and is being explored for potential use in humans.
Scientists discovered a protective variant of the HAQ-STING gene that prevents COPA Syndrome. This finding opens the door to a new gene therapy for the condition, which currently has no cure.
A novel bone marrow transplant process has been shown to be safe and curative for adults with sickle cell disease, offering a viable alternative to recent gene therapy products. The treatment, which uses a 'half-matched' donor, results in high cure rates and low side effects, making it a more accessible option for patients.
A new genetic medicine has been developed to treat a rare genetic deficiency affecting the AIPL1 gene, causing severe retinal dystrophy. The treatment involves injecting healthy copies of the gene into the retina through keyhole surgery, resulting in dramatic improvements in sight for four young children.
Researchers have discovered a novel gene therapy that can reverse conduction slowing and prevent cardiac arrhythmias by introducing the SCN10a-short gene into heart muscle cells. The treatment has shown promise in animal models and human cell studies, offering a potential solution for millions affected by arrhythmias worldwide.
Researchers at Virginia Tech have developed a method to convert gut bacteria into mini protein factories that produce and release sustained flows of targeted proteins within the lower intestine. This approach eliminates a major roadblock in delivering drugs to this part of the body, offering potential treatment for chronic diseases.
A new gene therapy has shown promise in treating a rare form of epilepsy, specifically Dravet syndrome, by replacing the SCN1B gene variant. The therapy increased survival rates, reduced seizure severity, and restored brain neuron excitability in mice with the condition.
The Rice University lab, in collaboration with Baylor College of Medicine, has developed a new gene-editing strategy called Repair Drive that improves the effectiveness of gene therapies in the liver. The technique enables the repair of liver cells at higher rates and equips them with a selective advantage to outcompete incorrectly edi...
Researchers at Virginia Tech have developed a way to convert gut bacteria into miniature protein factories that produce and release targeted proteins inside the lower intestine. This breakthrough could potentially treat chronic diseases.
Researchers discovered that mismatch repair genes are critical in eliciting damages to neurons vulnerable to Huntington's disease, triggering downstream pathologies and motor impairment. Targeting these genes may offer novel therapeutic approaches, including improving locomotor and gait deficits and reducing neuronal cell death.
Researchers at Osaka Metropolitan University assessed target genes in canine hepatocellular carcinoma (HCC) to develop molecular targeted therapies. The study identified potential gene targets, including PDGFB, which may improve treatment options for unresectable HCC.
Dr. Eric Levine's lab at UConn School of Medicine has been awarded $400,000 to study the genetic pathways of autism spectrum disorders using patient-derived human neurons. The team aims to identify genes and brain cell behavior that may cause these disorders, with potential applications for gene therapy.
Researchers developed a novel AAV-equipped nanomachine that successfully overcame gene therapy challenges in mice, including reduced efficiency due to neutralizing antibodies and hepatotoxicity. The nanomachine demonstrated sufficient gene transfer activity and suppressed liver toxicity markers.
A new study adds weight to the safety and effectiveness of a gene therapy for hypophosphatasia, a rare inherited disorder that causes abnormal bone development. The treatment, AAV8-TNAP-D10, has shown promising results in mice models, with female mice achieving improvements in bone and teeth at lower doses.
Researchers have developed a new gene therapy that targets aggressive brain cancer, glioblastoma, with a precise delivery system. The treatment uses a novel virus to deliver a targeting drug to cancer cells, achieving cure rates of up to 90% in mouse models.