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.
A new study identifies optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to treat Hemophilia A. The researchers successfully improved the design of AAV vectors, demonstrating significant and differing effects on liver-specific expression of the human factor VIII transgene in mice.
Researchers developed a gene therapy that restored balance and hearing in genetically modified mice with Usher Syndrome. The study's positive results suggest potential for treating various inherited vestibular and hearing disorders in humans.
Researchers have created a new cellular model to test the efficacy of new gene therapies for Chronic Granulomatous Disease more efficiently, using Crispr/Cas9 technology. The improved method allows for faster and cheaper development of new treatments.
Researchers envision AAV as a platform for delivering novel tools for genetic manipulation, including CRISPR-Cas9 and RNA interference. The combination of tailored delivery vectors and new editing techniques will enable unique approaches to therapeutic gene expression.
The Muscular Dystrophy Association (MDA) and the Charcot-Marie-Tooth Association (CMTA) have awarded a $119,999 grant to study gene therapy for CMT1X. The two-year research project aims to examine the effectiveness of repeated injections and treatment at later stages of the disease.
A recent study suggests that hormone replacement therapy may help improve heart health and overall survival in women, with lower levels of atherosclerosis and plaque buildup detected. The research found significant age-related benefits from the therapy, with women using it being 30% less likely to die than those not on hormone therapy.
A new study published in JOSPT found that physical therapy achieved results comparable to surgery outcomes for patients with carpal tunnel syndrome. Patients treated with physical therapy showed faster improvements and similar benefits at one year following treatment.
The St. Jude gene therapy program will deliver gene therapy to patients with X-linked severe combined immunodeficiency disease (X-SCID), also known as 'bubble boy' disease, at the University of California, San Francisco. The new grant supports widening access to this life-saving treatment for rare but deadly immune disease.
A recent study published in Neurology found no association between hormone therapy and the risk of developing Alzheimer's disease. However, women who used hormone therapy for over 10 years showed a reduced risk of Alzheimer's disease compared to non-users.
Researchers develop gene therapy to treat myotubular myopathy, a rare inherited disease causing fatal muscle wasting. A single infusion restores normal health in affected dogs, demonstrating potential for treating skeletal muscle disorders.
A new gene therapy has demonstrated effectiveness in clearing glycogen buildup from muscles in mice, potentially replacing enzyme infusions. The therapy uses a modified virus to deliver a gene that produces an enzyme missing in people with Pompe disease.
Scientists at Harvard Medical School have developed a new gene-delivery therapy that successfully restores partial hearing and balance in mice born with genetic hearing loss. The treatment uses a modified adeno-associated virus (AAV) wrapped in protective bubbles to penetrate hair cells, which are notoriously difficult to treat.