Researchers at CNIO develop gene therapy with telomerase, proving effective in mice against diseases caused by excessive telomere shortening. The study finds that the gene therapy does not increase the risk of developing cancer, even in a cancer-prone setting.
Gene therapy using optimized AAV to deliver human factor VIII showed substantial hFVIII expression and no detectable antibody response in cynomolgus macaques. The results suggest that AAVhu37-based gene therapy has the potential to advance to clinical trials for treating hemophilia A.
Scientists have developed a novel gene therapy that effectively reduces rhodopsin production and prevents photoreceptor death in dogs with autosomal dominant retinitis pigmentosa. The treatment, which combines shRNA interference with a replacement gene, shows promise for slowing or preventing vision loss in humans.
A study found significant improvement in cone function and no abnormalities in treated eyes for up to six years. The treatment used an AAV5 vector delivered a CNGA3 gene therapy, offering hope for treating genetic blindness.
A new study shows that a single injection of AAVB1-GAA gene therapy prolonged survival and improved enzyme activity in a mouse model of Pompe disease. The therapy also targeted the respiratory system, improving ventilatory measures.
The Alliance for Cancer Gene Therapy (ACGT) is funding innovative clinical approaches to find gene therapy treatments for solid cancers. Recipients of the 2018 research grants include a team working on a vaccine for melanoma, an injectable genetic agent to program T cell receptors, and CAR T cell therapy for brain cancer.
A new study published in Nature Medicine found that fetal gene therapy can prevent and cure neonatal lethal neurodegenerative diseases in humans in utero. The therapy was tested on mice with Gaucher disease and showed promising results, including improved brain degeneration and increased survival time. Researchers believe this approach...
The publication demonstrates the company's technology induces efficient and precise in vivo gene editing using homologous recombination, a natural DNA correction pathway. This early academic research translated into a scalable process for genetic medicines development.
Researchers from UAB successfully used gene therapy to treat type 2 diabetes and obesity in mice by inducing the production of FGF21, a hormone that maintains correct energy metabolism. The treatment led to weight loss and improved insulin sensitivity, reducing the risk of related comorbidities.
Researchers at Washington University School of Medicine have developed a gene therapy method to target damaged kidney cells, which could lead to improved treatment for chronic kidney disease. The approach uses adeno-associated virus (AAV) to deliver genetic material to targeted cells, showing promise in slowing or reversing cell damage.
A new molecular imaging method allows early assessment of gene therapy success, potentially improving treatment for Parkinson's and Alzheimer's diseases. The PET reporter gene/probe system enables noninvasive monitoring of gene expression in all brain areas.
A new gene therapy has been shown to restore hand function in rats with spinal cord injuries by breaking down scar tissue and allowing nerve cell regeneration. The therapy uses a 'stealth gene' switch that can be turned on and off, providing a safeguard and paving the way for human trials.
Researchers compared six adeno-associated viral (AAV) vectors for efficiency of gene transfer to both retinal pigment epithelial and photoreceptor cells. Human pluripotent stem cell-derived organoids were used as an in vitro test system for AAV vector development.
A genetic 'dial' controlling body size has been discovered in pigs, with decreased size observed at varying levels of gene expression. The study found that pigs with normal gene expression were average-sized, while those with one copy expressed had a 25% reduction and those without any expressed had a 75% reduction.
A gene therapy case report published in Human Gene Therapy reveals a safe and effective treatment for lipoprotein lipase deficiency, with improved quality of life outcomes after discontinuing plasmapheresis. The treatment, ali-pogene tiparvovec, has been approved in Europe to treat the rare inherited disorder.
Researchers at Duke University successfully delivered CRISPR/Cas9 repressors to silence the Pcsk9 gene, which regulates cholesterol levels, in adult mice. The treatment resulted in reduced blood cholesterol levels and sustained gene repression for six months after a single treatment.
A new gene therapy has successfully treated transfusion-dependent thalassemia, a blood disorder that requires frequent red blood cell transfusions. The treatment produced positive outcomes in an interim analysis of two international clinical trials, with most patients becoming transfusion-free.
A naturally occurring gene variation affecting women's body fat distribution puts them at significantly higher risk of type 2 diabetes. The discovery could lead to the development of drugs targeting this specific gene variation.
Researchers found that gene therapy using adeno-associated viral vectors can only temporarily alleviate congenital adrenal hyperplasia (CAH) by replacing the defective gene. A long-term solution requires targeting genetic mutations in adrenocortical stem cells, according to a study published in Human Gene Therapy.
A new study in mice shows that turning on a gene called LZK can stimulate the healing process after spinal cord injuries, resulting in smaller scars. This trigger has implications for treating brain conditions through gene therapy targeting astrocytes.
Therapy animals are used in mental and physical health treatment, but lack regulation; guidelines aim to prevent misuse and misunderstanding. The new recommendations include standard terminology, clear methodologies, peak bodies, and ongoing research.
A special issue of Human Gene Therapy highlights Chinese research advancements in gene and cell therapy, including novel viral and nonviral vectors for gene delivery. The articles explore innovative applications of CRISPR technology in various disease areas.
Researchers at the German Primate Center have improved DNA transfer in gene therapy by modifying viruses to produce a protein called CD9, which increases exosome production and virus efficiency. This results in an 80% infection rate, making gene therapies more efficient.
Researchers at Osaka University have developed a novel CRISPR-Cas9 method that can introduce precise modifications to defective genes with fewer safety drawbacks. The new technique, called Single Nicking in the target Gene and Donor (SNGD), reduces unintended genetic mutations by up to 95%.
A global, multicenter study has found CAR T-cell therapy to be safe and effective in treating patients with relapsed or refractory B-cell ALL. The therapy achieved an overall remission rate of 81% within 3 months of treatment, with a median length of remission lasting up to 20 months.
Researchers found liver and neuronal toxicity with high doses of gene therapy delivered using an adeno-associated virus (AAV9) vector. The study warns about the importance of prioritizing patient welfare in gene therapy research.
A new viral gene therapy delivers extra copies of the SOD2 and CTGF genes to protect healthy tissues from damage caused by radiotherapy. The treatment has shown promising results in preventing tissue damage and scarring in rats treated with radiotherapy, potentially improving breast reconstruction outcomes for women with breast cancer.
Researchers are exploring gene delivery strategies for osteoarthritis and rheumatoid arthritis, enabling sustained drug production at the site of disease. Clinical trials are underway in the U.S., with the first arthritis gene therapy recently approved in Korea.
Recent advances in gene therapy have led to successful treatments for patients with serious medical conditions. Emerging genome editing technologies, such as CRISPR/Cas9, hold promise for broader and more effective gene therapy approaches.
Researchers have identified a specific gene, ADCY3, that predisposes Greenlanders to obesity and diabetes when it is inactive. The study, published in Nature Genetics, found that 4.4% of test subjects had the gene inactive, leading to significant weight gain and increased risk of diabetes.
A groundbreaking gene therapy trial has shown that over one year on from a single treatment, participants with haemophilia A are showing normal levels of the previously missing protein, effectively curing them. The results have particular significance as the first successful gene therapy trial for the haemophilia A.
Researchers found a shift in cytokine profiles of mice with FVIII inhibitor development, including increased levels of pro-inflammatory cytokines. A negative correlation was also observed between risk factors for FVIII inhibitor development and age at gene therapy administration.
A new gene therapy has successfully produced sustained levels of clotting factor in adult hemophilia B patients, eliminating the need for regular infusions. The treatment, which uses a highly active variant of the clotting factor, achieved significant clinical benefits with no adverse effects.
A clinical trial suggests a new form of gene therapy, targeting CD22, achieved significant remission rates in children and young adults with treatment-resistant B-cell leukemia. The therapy was well-tolerated and showed promise in patients who had previously failed anti-CD19 CAR T-cell treatment.
Researchers developed a novel gene therapy that lasts for over eight months, reducing methamphetamine in the brain and its stimulant effects. The therapy generates antibodies against meth, preventing it from reaching the brain.
RefEx is a web tool for comfortable search of reference data in gene expression analysis. It offers extensive collection of gene expression data from human, mouse, and rat tissues/cells measured by four different methods, enabling comparison of gene expression status among tissues/cells and measurement methods.
A Phase 1 clinical trial has demonstrated encouraging data for gene replacement therapy in patients with spinal muscular atrophy type 1 (SMA1). The study found that high-dose gene therapy improved motor function and reduced the need for supportive care in patients treated with a single intravenous infusion.
A new Northwestern Medicine study shows proton therapy outperforms intensity-modulated radiation therapy (IMRT) in five-year overall survival rates, with a 93.25% rate for proton patients compared to 88.43% for IMRT patients. Proton therapy also reduces complications and secondary malignancies, particularly for younger patients.
A study found that proton beam therapy in combination with chemotherapy prior to surgery reduces postoperative heart and lung problems and mortality rates in elderly patients. Researchers followed 571 patients treated with traditional radiation therapy and chemotherapy followed by surgery at Mayo Clinic Cancer Center.
German researchers have made significant advancements in human gene therapy, including virotherapy capable of destroying tumor cells and engineered hematopoietic stem cell delivery systems. These innovations hold promise for treating immunodeficiencies and genetic diseases.
Researchers at Alliance for Cancer Gene Therapy (ACGT) have received $1.3 million in critical funding to study immunotherapy and virotherapy for three deadly forms of cancer: glioblastoma, sarcoma, and ovarian cancer. The grants will support clinical trials to test innovative treatments using gene therapy.
A study published in JAMA found that insulin pump therapy among young patients with type 1 diabetes was associated with lower rates of severe hypoglycemia and diabetic ketoacidosis compared to traditional injections. This innovative treatment approach offers improved clinical outcomes for this vulnerable population.
Researchers successfully built a gene drive to reduce female fertility in malaria-spreading mosquitoes, but mutations soon arose that blocked the spread of new genes. This study documents the emergence of resistance and will help improve future gene drives.
A new study successfully uses a shortened version of the CEP290 gene to treat Leber congenital amaurosis type 10, a rare genetic disorder causing blindness. The treatment shows significant improvement in photoreceptor survival and function.
Researchers report that a gene therapy to treat cerebral adrenoleukodystrophy has effectively stabilized the disease's progression in 88 percent of patients. Fifteen out of 17 patients had stable neurologic functioning more than two years after receiving the treatment.
Researchers used gene therapy to improve heart function in a large animal model, finding a 25% improvement in left ventricular function and a 20% improvement in left atrial function. The treatment also reduced heart size by 10%.
Researchers at Institut Pasteur successfully restored hearing and balance in a mouse model of USH1G syndrome using gene therapy, offering new hope for the treatment of hereditary deafness. The study demonstrates that inner ear defects caused by mutations in the USH1G gene can be reversed even after birth with durable efficacy.
Researchers used gene therapy to stop the immune response that causes multiple sclerosis in mouse models, producing near-complete remission. The treatment combined a brain-protein gene with an existing medication, showing significant potential for treating multiple sclerosis and other autoimmune disorders.
A new study uses gene transfer to block the expression of an enzyme that breaks down alcohol, leading to increased acetaldehyde levels and potential treatment for alcoholism. The approach has strong proof-of-concept data and bodes well for future development of a genetic therapy.
The FDA has approved Kymriah, a CAR T gene therapy drug, for pediatric and young adult patients with ALL, marking a major milestone in cancer treatment. The therapy showed an exceptional 83 percent success remission rate in clinical trials.
Researchers at the University of Chicago have developed a novel gene therapy approach that uses skin transplants to treat type-2 diabetes and obesity. The method uses CRISPR technology to deliver a hormone that stimulates insulin production, reducing blood glucose levels and promoting weight loss.
Researchers developed a gene therapy using microdystrophin to treat Duchenne muscular dystrophy in dogs. The treatment restored muscle function and stabilized clinical symptoms for over 2 years. This breakthrough could lead to a new treatment for children with the disease.
Researchers found that neoadjuvant radiation therapy significantly lowers the risk of developing a second primary tumor in early-stage breast cancer patients. The study also suggests that delaying surgery due to neoadjuvant radiation therapy is not detrimental to survival.
The FDA advisory committee voted unanimously to recommend approval of Novartis' CAR-T therapy Tisagenlecleucel, demonstrating impressive results in hard-to-treat leukemia patients. ACGT's funding played a crucial role in advancing the pioneering treatment.
Scientists at UC Berkeley and UC Riverside have developed a gene editing technique using CRISPR/Cas9 technology to suppress mosquitoes carrying diseases like malaria. The new technique, multiplexing, can target multiple locations in a gene simultaneously, increasing the effectiveness of gene drive systems.
A new study found that oral prednisone reduced immune responses to AAV-based gene delivery, resulting in a 60% decrease in cytotoxic T cell infiltrates. The treatment also increased PD-L2 levels, which induce programmed T-cell death, suggesting a potential therapeutic benefit for gene therapy.
Recent clinical trials using different gene transfer approaches have shown acceptable risk and potential for treating heart failure with reduced ejection fraction. Despite mixed results in terms of efficacy, the data support further clinical development of this therapeutic strategy.
Researchers used CRISPR genome editing technology to target unique DNA sequences formed by fusion genes, cutting out mutated DNA and replacing it with a gene that leads to cancer cell death. This approach improved survival rates in mouse models of aggressive liver and prostate cancers.
A new review discusses how gene therapy delivered via adeno-associated viruses (AAV) can trigger an immune response in T cells, leading to activation and exhaustion. The review highlights the variability in individual patients' immune responses to AAV vectors and potential strategies to suppress these reactions.
A growing number of preclinical studies suggest that rAAV-mediated gene therapy may cause insertional mutagenesis and increase the risk of hepatocellular carcinoma. Research emphasizes the need for careful reconsideration of potential genotoxicity risks in human clinical applications.