Articles tagged with Gene Therapy
A study published in Proceedings of the National Academy of Sciences successfully reversed hearing loss in mice by activating a defective gene. The researchers found that introducing a special enzyme at a young age significantly improved hearing, with positive effects becoming less potent with time.
Researchers have made a significant discovery in gene therapy, showing that genetically engineered blood stem cells can be created while still in the bone marrow. This approach eliminates the need for chemotherapy and transplantation of corrected cells, cutting costs and improving access to critical treatments.
Researchers at NYU College of Dentistry developed a gene therapy that regulates sodium ion channel activity to alleviate chronic pain. By targeting the specific region where CRMP2 binds to NaV1.7, they were able to reduce sodium influx and quiet down neurons, providing relief from pain in cell and animal studies.
A team from Tokyo Medical and Dental University has developed a technique to improve bone regeneration over large areas in rats, using vascular endothelial growth factor (VEGF) and Runx2. The combination of these two RNAs led to better regenerative responses in bone cells than each RNA alone.
A team of scientists at Harvard Medical School has identified six chemical cocktails that can restore cellular aging and rejuvenate human cells. The study builds upon the discovery of Yamanaka factors, which can convert adult cells into induced pluripotent stem cells, raising hopes for treating age-related diseases and injuries. The im...
Hematopoietic stem cell culture technology improves genome editing in HSCs by increasing successful correction rates to 100%, eliminating genetic mutations, and enhancing cell transplantation outcomes. This breakthrough enhances the efficiency and safety of gene editing in treating genetic diseases.
A team of researchers has identified a unique genetic signature in CAR T-cells that enables them to persist in the body for a longer time, leading to improved remission rates for children with leukaemia. This discovery provides a new understanding of why some CAR T-cells last longer and can help improve treatment outcomes.
Researchers developed a new method to deliver drugs into the inner ear, using the cerebrospinal fluid flow. This innovation restored hearing in deaf mice and may pave the way for gene therapy to treat human hearing loss.
Researchers at MD Anderson Cancer Center have identified disparities in end-of-life immunotherapy treatment, highlighting the need for further examination to ensure quality care. A new study also reveals a novel target to improve immunotherapy responses in KRAS-mutant lung cancer and strategies to manage immune-related toxicities.
Researchers at MD Anderson Cancer Center have engineered a new model of aggressive renal cell carcinoma, highlighting molecular targets and genomic events that trigger chromosomal instability. The loss of interferon receptor genes plays a pivotal role in allowing cancer cells to become tolerant of chromosomal instability.
Researchers at Tel Aviv University have developed an innovative gene therapy that shows promise in treating Dravet syndrome, a severe developmental epilepsy affecting children. The treatment was found to be effective in improving epilepsy, protecting against early death, and enhancing cognitive abilities.
New research suggests optogenetics could restore regular heart rhythm without shocks, improving prognosis and quality of life for AF patients. Light therapy has shown promising results in rats, indicating potential translatability to humans.
Researchers at Washington University School of Medicine developed a catalog to classify protein variants associated with limb girdle muscular dystrophy. The approach enables doctors to determine the pathogenicity of variants, leading to more precise diagnoses and potential therapies.
Indiana University School of Medicine researchers will investigate muscle-directed gene therapies and test alternative treatment options for degenerative disorders like Duchenne muscular dystrophy. The goal is to develop more successful and long-term ways to help patients living with muscle disorders.
A single dose of anti-Müllerian hormone gene therapy can induce long-term contraception in female domestic cats by suppressing ovarian follicle development and ovulation. The treatment maintained high AMH levels for over two years, demonstrating its safety and effectiveness.
Researchers identified mRNAs and long non-coding RNAs targeted by stress granule proteins, which accumulate AD-associated gene transcripts in these structures. SGs may play a key role in regulating AD development through the impairment of protein neurohomeostasis.
Researchers discovered that ZC3H11A is essential for regulating metabolic genes in embryos and their absence leads to complete lethality. The study's findings suggest that ZC3 may be a promising therapeutic target for the development of anti-viral agents against medically significant human viruses.
An observational study of 329,000 Medicare admissions found that older persons receiving hospital care from allopathic (M.D.) or osteopathic (D.O.) physicians experience similar quality and cost of care. Researchers also highlight systemic health inequities faced by persons with sickle cell disease.
Researchers successfully demonstrate AAV vector efficacy in aged animal models, showing robust hearing rescue in mice with a mutation equivalent to a defective human gene. The study suggests that virally mediated gene therapy could potentially treat genetic hearing loss, especially for patients diagnosed at advanced age.
Researchers have discovered that HER3 plays a crucial role in promoting cell survival in metastatic colorectal and pancreatic cancer. The surrounding liver microenvironment activates HER3, making it an emerging therapeutic target for these types of cancer.
A new study demonstrates that subretinal delivery of AAV-RPE65 gene supplementation increases RPE65 isomerase activity and recovers chromophore 11-cis retinal after bleaching in mice with D477G RPE65 mutation, providing initial proof-of-principle data for gene supplementation as a treatment.
Gene therapy is being tested for rare skeletal dysplasia, affecting 1 in 200,000. Patients will receive a one-time infusion of viral vector containing correct gene sequence.
Increased expression of Musashi 1 on breast cancer cells has significant implications for understanding dormancy and survival in bone marrow. Msi 1 knockdown led to a reduction in cancer stem cells with undetectable PD-L1, suggesting a potential therapeutic target.
Researchers used cancer proteomics data to identify gene candidates for therapeutic targeting, focusing on protein kinases in uterine endometrial cancer cells. Public molecular resources and multi-omics data analysis can prioritize genes of interest for future studies.
Researchers developed a size exclusion chromatography (SEC) method to measure empty capsids in gene therapy programs, outperforming other analytical methods. The study found SEC-MALS to be linear, accurate, and precise, providing a robust analytical technique for advancement of gene therapy programs.
The LY6 gene family has been found to be overexpressed in uterine corpus endometrial carcinoma (UCEC), leading to poor patient survival. Several LY6 genes have been identified as potential tumor-associated antigens and biomarkers for UCEC detection and prognosis.
Scientists have developed a new method to deliver genetic information to stem cells using nanoparticles coated with a specific polymer, enabling more efficient control over cellular differentiation. This innovation has the potential to improve the efficiency and effectiveness of regenerative medicine treatments.
Researchers identified three novel dual-purpose therapeutic targets using PandaOmics, which could treat both aging and glioblastoma multiforme. The target hypotheses include cyclic nucleotide gated channel subunit alpha 3 (CNGA3), glutamate dehydrogenase 1 (GLUD1) and sirtuin 1 (SIRT1).
Researchers at Brigham and Women's Hospital have identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma. MicroRNA nanoparticles delivered locally using a hydrogel suppressed osteosarcoma growth while decreasing bone damage.
Researchers evaluated six AAV vectors in 12 preclinical liver models to identify the most relevant combination for safe and efficient gene therapy. The study proposes using multiple models to provide a more complete picture of vector function, informing clinical decisions.
Researchers successfully delivered stabilized divalent siRNA molecules to animal models that blocked SARS-CoV-2 and prevented infection. The technology is adaptable for other pulmonary diseases such as pulmonary fibrosis and respiratory viruses.
A new therapy delivery method, using modified viruses engineered with fusogens Myomaker and Myomerger, shows promise as a treatment for Duchenne muscular dystrophy. The vector can deliver a vital gene needed for muscle function to cells, potentially providing a lifelong supply of the missing gene.
Scientists design a mini gene therapy to replace the mutated gene in Usher 1F, increasing protein production and restoring hearing in mice. The approach may also prevent blindness by targeting a different form of the protein involved in vision loss.
A new genetic therapy, BIIB080, has been shown to safely lower levels of the harmful tau protein in patients with Alzheimer's disease. The trial found a greater than 50% reduction in tau protein concentrations after 24 weeks in treatment groups.
Researchers have developed a bioengineered AAV3B capsid variant with improved transduction to human liver cells and reduced seroreactivity to human serum samples. The variant, AAV3B-V04, demonstrates enhanced hepatocyte tropism and immune evasion, making it a promising candidate for gene therapy.
Cedars-Sinai investigators have discovered a novel way to treat amyotrophic lateral sclerosis (ALS) and retinitis pigmentosa using human induced pluripotent stem cells. The new approach uses cells derived from iPSCs that are renewable, scalable, and can delay disease progression in rodents.
A novel gene therapy approach decreases intraocular pressure in pre-clinical models of glaucoma. The treatment uses a viral vector to deliver instructions to cells, producing an enzyme that helps reduce fluid accumulation and pressure. This breakthrough holds promise for patients with glaucoma.
Researchers have created a drug-like cocktail that successfully regenerated hair cells in a mouse model by reprogramming genetic pathways within the inner ear. The approach could lead to clinical trials for a gene therapy that can be administered to people with hearing loss, potentially treating up to 90% of cases.
Researchers have built a new model to examine Usher Syndrome, a leading cause of combined deafness and blindness. The model replicates the visual problems not addressed by previous models, offering insight into strategies for designing therapeutic interventions.
Researchers at Tokyo Medical and Dental University have found that a specific lipid, alpha-tocopherol, increases the uptake of antisense oligonucleotides in the stroke-lesioned brain. This delivery method has potential for targeted protein expression after a stroke.
Researchers at the University of Tokyo have discovered the 3D structure of TnpB, a protein involved in genome editing and a probable precursor to the CRISPR-Cas12 enzyme. The study reveals how TnpB recognizes and cuts DNA using a unique pseudoknot shape similar to that found in guide RNAs of Cas12 enzymes.
Researchers at Indiana University School of Medicine investigated the protein BVES and its crucial role in muscular dystrophy. They discovered that inhibiting proteasome degradation with Bortezomib can alleviate symptoms of BVES-deficient muscular dystrophy.
Researchers at Children's Hospital of Philadelphia discovered that viral proteins use phase separation to coordinate the complex process of replicating viral genomes and then encapsulating them in a viral particle. This process allows for the orderly and coordinated formation of infectious viral offspring.
A recent study found that a tiny fragment of the PC1 protein can suppress the disease in mouse models, reducing cyst size and revealing new avenues for developing therapies. The researchers discovered that this small piece interacts with a mitochondrial protein called NNT, opening up new avenues to study its normal function.
Researchers developed a programmable bacterial injection system that can deliver a range of proteins, including those for gene editing, to different cell types. The system has shown promising results in targeting cancer cells and delivering proteins to the brain in live mice without provoking an immune response.
The NCATS Platform Vector Gene Therapy (PaVe-GT) program utilizes FDA guidance to prepare applications for Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD), stimulating gene therapy development. The PaVe-GT program has already helped receive ODD and RPDD designations for a gene therapy product treating PCCA-r...
Researchers at Tokyo Medical and Dental University have developed a polymeric nanoparticle gene delivery system that promotes bone formation after traumatic inflammation. The therapy inhibits excessive inflammation and prevents residual ridge resorption, leading to improved tissue healing after tooth extraction.
Researchers have found that valosin-containing protein (VCP) is essential for KRAS-mutant pancreatic ductal adenocarcinoma cell growth and survival. Inhibiting VCP, combined with autophagy inhibition, enhances efficacy in preclinical studies.
Researchers used a monoclonal antibody to reduce circulating IgG levels, allowing for successful gene delivery to the liver and heart via systemic AAV-based gene therapy in mice and non-human primates. The strategy has potential to circumvent neutralizing antibody limitations and expand gene therapy accessibility.
Researchers at Texas A&M University have developed the first molecular therapeutic for Angelman syndrome, a devastating neurogenetic disorder. The therapy targets an evolutionarily conserved region in the UBE3A-AS transcript and has shown promising results in clinical trials.
Researchers at Harvard University developed a novel RNA sense-and-respond circuit, DART VADAR, which utilizes an enzyme to detect specific molecular markers of disease and cell types. This enables highly specific treatments for various diseases by triggering the translation of therapeutic genetic payloads.
Researchers at UCLA successfully used base editing to correct a mutation causing rare immune deficiency CD3 delta SCID. The treatment corrected an average of 71% of patient stem cells and allowed them to produce fully functional T cells, suggesting long-term persistence of corrected blood stem cells.
A new study published in Blood Advances found that the average life expectancy of publicly insured individuals with sickle cell disease is roughly 52.6 years, which is significantly lower than the overall US life expectancy of 73.5 years for men and 79.3 years for women. The study also revealed worse survival outcomes among those insur...
Researchers have designed a new RNA sensor that can selectively activate synthetic genes in specific cells, opening up possibilities for targeted therapies for cancer and other diseases. The system uses an enzyme that naturally exists in most animal cells to detect and repair mismatches in double-stranded RNA.
Researchers have successfully used AAV1.NT-3 gene therapy to improve muscle physiology and prevent age-related sarcopenia in mice. The treatment resulted in restored muscle mass, strength, and neural connections, offering a potential new option for managing this debilitating condition.
Pancreatic cancer is an incurable form of cancer, but gene therapies are being developed to treat it. The article reviews ongoing clinical trials and discusses various biotherapies, including vaccines and CAR-T cell therapy.
Gene therapy using CRISPR-Cas9 lipid nanoparticles has been shown to be highly effective in reducing target protein expression in mice. The new delivery system increases the efficiency of in vivo gene therapy, paving the way for safe and effective treatment.
Researchers found that severe herpesvirus infections can strongly activate host cellular immunity, leading to a therapeutic effect on refractory adult T-cell leukemia/lymphoma. This activation may play an important role in the survival of patients with this intractable disease.
Researchers have developed a new gene therapy to treat alpha 1-antitrypsin deficiency, an autosomal recessive disorder that causes emphysema. The therapy uses an adeno-associated virus (AAV) vector coding for an oxidation-resistant human α1-antitrypsin protein.
Researchers at UTHSC are working on a project to find the first therapeutic intervention to prevent frontotemporal dementia or slow its progression in a mouse model linked with the condition. They aim to use DNAzymes to target pathological tau aggregates, which cause cognitive impairment and progressive neuropathological symptoms.