Researchers from Mass General Brigham presented key findings from multiple innovative studies on gene and cell therapy, focusing on rare diseases, brain cancer, and neurodegenerative disorders. The studies explored strategies to improve care delivery and accelerate translation from lab to clinic, with potential breakthroughs in treatin...
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.
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 new machine learning model accurately predicts the fitness of AAV capsids based on their amino acid sequence, enabling more efficient and cost-effective gene therapies. The model's robustness and generalizability have been demonstrated through tests on independent datasets, offering a promising tool for capsid engineering.
Swim Across America has awarded two $450,000 grants to support novel gene and base editing techniques in advanced cancer research. These grants will accelerate the development of potential gene-edited cancer treatments that could transform how we treat patients.
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.
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 developed tomoseqr, a user-friendly software to estimate 3D spatial gene expression distribution. The software successfully reproduced known gene expression patterns and mapped the 3D spatial distribution of genes in zebrafish and planarians.
A new study found that recombinant adeno-associated virus (rAAV) capsids contain single-stranded DNA impurities derived from plasmid and host cell DNA. The researchers suggest that the adverse effects of these impurities may differ from those of double-stranded DNA, highlighting the need for further evaluation.
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.
A research team from UH Seidman Cancer Center developed an ultra-fast and highly scalable CAR T-cell therapy manufacturing platform, enabling wider utilization of the therapy. The approach resulted in significantly more favorable toxicity profiles compared to traditional CAR-T products.
A study published in Human Gene Therapy found that over half of individuals with Niemann-Pick disease type C1 lacked neutralizing antibodies against AAV2 and AAV9. This absence of antibodies may impact the effectiveness of gene therapy treatments for this rare disorder.
Professor Robert T. Schooley will present a talk at the 8th World Congress on Targeting Phage Therapy, exploring phage therapy advancements and necessary steps for widespread adoption. The congress will gather experts to discuss latest advances, challenges, and clinical applications of bacteriophage therapy.
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 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 new gene therapy has been trialled in humans with cystic fibrosis, aiming to improve lung function and reduce exacerbations. The treatment, BI 3720931, works by inserting a functioning copy of the CFTR gene into patients' airways.
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.
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.
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.
A recent gene therapy study for advanced metachromatic leukodystrophy (MLD) showed stabilization or improvement in two patients, but lacks critical clinical assessments. The research highlights the need for comprehensive evaluations of cognition, motor, and speech function to substantiate claims.
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.
A study by National Institute of Standards and Technology scientists has highlighted the need for standardized measurement methods in gene therapy. The researchers evaluated four techniques used to measure modified viruses deployed in some gene therapy research and treatments, finding that one technique had poor accuracy and precision.
Researchers developed a gene therapy approach to treat chronic hypereosinophilia by delivering an anti-human eosinophil antibody via AAV-based gene therapy. The therapy successfully suppressed blood eosinophil levels in mice, showing promise as a potential treatment for the condition.
A new gene therapy has reversed the effects of heart failure in a large animal model by increasing blood pumping efficiency and dramatically improving survival rates. The therapy restored critical functions of heart cells and improved heart function on the microscopic level.
A new study found that gene therapy delandistrogene moxeparvovec significantly extended the median survival of Duchenne muscular dystrophy (DMD) rats to >25 months. Additionally, the treatment elicited statistically significant improvements in cardiac parameters and mobility.
A Phase 2 clinical trial has been initiated by Genethon and Hansa Biopharma to evaluate the efficacy and safety of imlifidase, a gene therapy for Crigler-Najjar syndrome. The trial aims to address anti-AAV antibodies, which prevent up to 1 in 3 people from benefiting from gene therapies.
Positive initial results from Genethon's gene therapy GNT0004 show stabilization of motor functions and improved dystrophin expression in patients with Duchenne Muscular Dystrophy. The therapy is expected to be launched in pivotal trial phases in Europe and the US in Q2/2025.
Researchers developed a novel lentivirus-based gene therapy strategy in CD34+ hematopoietic progenitor cells, which showed therapeutic levels of expression of the anti-sickling beta globin protein. Cyclosporin improved transduction efficiency and preserved cell viability.
The study found that high oxygen therapy modalities increased oxidative stress and lung injury in patients with severe COVID-19 pneumonia. Patients who died of COVID-19 had significantly higher mean MDA levels than those who survived.
A new RNA-based technology called StitchR facilitates effective use of gene therapy for difficult-to-treat, large-gene diseases like muscular dystrophies. It delivers two halves of a gene separately, resulting in seamless reconstitution of large mRNA in affected tissues.
The institute's Spark Grant program recognizes innovative research in gene and cell therapy, with recipients working on projects spanning disease fields such as neuroinflammation, cancer, and tuberous sclerosis. The $1.15M grant will support the development of novel treatments and commercialization outcomes.
The new textbook, Gene Regulation and Epigenetics: How Science Works, provides an in-depth understanding of gene regulation and epigenomics, essential for graduate students across biomedical fields. The book highlights the role of transcription factors, chromatin dynamics, and non-coding RNA in shaping gene expression.
The US Department of Defense has awarded $514,000 to study the efficacy of gene editors in treating Duchenne Muscular Dystrophy. Researchers will explore non-viral options for delivering gene therapy through 'self-delivering' gene editors, aiming to improve safety and efficacy.
Using state-of-the-art technology, researchers identified specific steps needed for CRISPR to become active and perform its gene editing function. Understanding these mechanisms could lead to improved designs for CRISPR-based gene editing.
Researchers have developed a novel gene therapy called PS-002, which targets podocytes and blocks complement activation to treat IgA nephropathy. In mouse models, treatment with PS-002 reduced signs of kidney dysfunction and lowered complement deposition, while in pigs, it resulted in elevated gene expression without safety issues.
Researchers used ex vivo lentiviral gene therapy to treat MPS IVA in mice, achieving partial correction of bone pathology and complete correction of heart pathology. The study suggests potential for novel therapies to treat patients with MPS IVA.
Researchers aim to improve gene therapy design for life-long correction of genetic diseases, but face unknowns including immune response and genomic changes. A five-year NIH award will fund an analysis of genetic and cellular determinants of gene therapy longevity.
Researchers at ChristianaCare Gene Editing Institute use CRISPR tools to safely disable gene mutation linked to treatment-resistant melanoma. The approach targets melanoma tumor cells while leaving healthy cells alone, restoring sensitivity to anticancer drugs.
Researchers have developed a novel gene therapy approach that targets and breaks down faulty ribonucleic acids in the KCNA2 gene, which is associated with recurring seizures. The therapy has shown promise in reducing excessive neuron activity linked to epilepsy.
A single-dose gene therapy has been shown to significantly reduce bleeding episodes in adults with hemophilia B, with an average reduction of 71% compared to standard treatment. The therapy, which enables the liver to produce clotting factor IX, has been FDA-approved for use in patients with this genetic disorder.
A new NIH-funded project aims to address gene therapy's ethical and policy challenges, gathering information from experts in six domains. The study will provide recommendations for addressing these challenges, with implications for patient safety and health outcomes.
Genethon has developed an innovative gene therapy vector that effectively targets muscle tissue while reducing the risk of liver penetration. The new capsid design uses AI predictive methodology to improve efficacy and safety, paving the way for more effective treatments for neuromuscular diseases.
Scientists from Spirovant Sciences describe a novel adeno-associated virus (AAV) gene therapy called SP-101 that has been optimized for efficient human airway cell transduction. After single dose inhaled delivery, the vector showed consistent expression of a functional and regulated shortened human CFTR minigene.
Children's Hospital of Philadelphia researchers have reported encouraging evidence of an effective new gene therapy to treat multiple sulfatase deficiency. The ex vivo gene therapy improved sulfatase production and reduced symptoms associated with the disease in preclinical models.
Scientists from Trinity College Dublin have developed a gene therapy that protects retinal ganglion cells and improves their function in animal models of glaucoma. The therapy has also been shown to increase oxygen consumption and ATP production in human retinal cells, indicating enhanced cell performance.
Researchers developed a gene therapy that restored useful vision to most patients with Leber congenital amaurosis type I, a rare inherited blindness. The treatment showed a 10,000-fold improvement in light sensitivity and improved navigation abilities in patients who received the highest dose.
The Rice University lab has developed a new noninvasive technique, called REMIS, that can measure gene expression and gene therapy delivery in specific brain regions using ultrasound. This technology could revolutionize brain-based gene therapy for neurodegenerative diseases.
Researchers developed a triple-adeno-associated virus vector system to deliver full-length dystrophin protein into muscles, restoring muscle health and strength in mice models with Duchenne muscular dystrophy. The approach has shown promise for treating patients with the genetic disorder.
Researchers found that subretinal adeno-associated virus 8 injections trigger proinflammatory T-cell responses, but co-injection of immunodominant peptides can modulate the immune system. This study suggests a new approach to AAV gene therapy for retinal diseases.
Researchers at UW Medicine have developed a new gene therapy that delivers protein packets to replace defective genes in muscles, halting disease progression and reversing pathology. The therapy uses adeno-associated viral vectors and aims to restore normal muscle health, with human trials expected to begin in two years.
Researchers used gene editing to restore hearing in adult mice with a form of inherited deafness called autosomal dominant deafness-50 (DFNA50). The approach involved shutting down a damaged copy of the microRNA (miRNA) gene, which enabled the animals to regain hearing. This method may eventually lead to potential treatments for inheri...
A trial published in The BMJ highlights the need to optimise speech therapy resources for people with Parkinson’s disease. LSVT LOUD is more effective than conventional NHS speech and language therapy or no therapy in improving voice handicap scores.
A new CAR-T cell therapy targeting CD22 has shown significant improvement in patients with large B-cell lymphoma, with over half experiencing a complete response. The therapy, which targets a different protein on the surface of cancer cells, has improved outcomes for patients who have failed previous treatments.
A groundbreaking clinical trial has shown that gene therapy can halt the progression of spastic paraplegia type 50 (SPG50) in a young boy. The treatment, delivered via spinal fluid, eliminated serious side effects and showed potential signs of improvement.
A first-of-its-kind study compared preventative therapy efficacy between two groups: gene therapy alone and in combination with risdiplam or nusinersen. Dual therapy showed promise in independent sitting outcomes, but not in walking age or muscle disease progression prevention.
Researchers developed an ex vivo gene-editing protocol to treat hereditary tyrosinemia type 1 using liver cell therapy. The study showed robust engraftment and expansion of transplanted gene-edited hepatocytes in recipient mice, correcting disease indicators such as normalized tyrosine, phenylalanine levels.
Scientists have improved a gene-editing technology that can insert or substitute entire genes in the genome, potentially treating multiple genetic diseases with a single therapy. The new approach, eePASSIGE, uses prime editors and recombinase enzymes to make gene-sized edits several times more efficiently than previous methods.
Researchers elucidated the spatial structure and molecular mechanisms of 'prime editor,' a novel gene-editing tool that achieves reverse transcription without DNA cutting. This breakthrough contributes to designing gene-editing tools accurate enough for gene therapy treatments, opening new avenues for both basic and applied research.