Articles tagged with Gene Therapy
Researchers have explored the potential of this novel therapeutic approach, demonstrating promising pre-clinical data with over 40 scientific papers. Vectored-antibody delivery using rAAV offers advantages in treatment of various chronic and infectious diseases.
Researchers at University of California San Diego use CRISPR/Cas9 to reprogram mutated rod photoreceptors into functioning cone photoreceptors, reversing cellular degeneration and restoring visual function in two mouse models of retinitis pigmentosa. The approach shows promise for advancing human clinical trials.
Dr. John Lahey has been honored with the first-ever ACGT Edward Netter Award for his contributions to cancer gene therapy. He is recognized for his strategic planning that grew student enrollment from 2,000 to nearly 10,000 students at Quinnipiac University.
Scientists develop a targeted therapy that slows disease progression in mice with ataxia and ALS, suggesting potential benefits for humans. The treatment targets the ataxin-2 gene, which may also be important for maintaining brain cell health.
Scientists at St. Jude Children's Research Hospital have successfully regenerated auditory hair cells in adult mice using genetic manipulation. The research marks a significant step towards treating hearing loss in humans, which affects millions worldwide.
Researchers from George Washington University have discovered a potential new drug to treat and stop the progression of cystic fibrosis. Thymosin α1 (Tα1) corrects genetic and tissue defects, reduces inflammation, and increases CFTR maturation, stability, and activity.
A team of researchers identified a genetic mutation responsible for an adult's rare and severe immune system disorder. They developed a molecular therapy using Morpholino Antisense Oligonucleotide that corrected the mutation and allowed the patient's immune system to function properly.
A new gene therapy approach has restored muscle strength and prolonged life in dogs affected by myotubular myopathy, a rare X-linked genetic disorder. The treatment, administered via a single intravenous injection, was developed by Genethon and demonstrated efficacy in a large animal model.
A recent study published in Biological Psychiatry has identified a link between rare variants of the NKPD1 gene and depressive symptoms. Researchers found that nearly 2,000 people with depressive symptoms were analyzed, revealing a possible genetic connection to depression.
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 at Brigham and Women's Hospital have discovered a new genetic variant linked to Tau pathology in Alzheimer's disease. The study, published in Molecular Psychiatry, highlights the potential for targeting the protein tyrosine phosphatase receptor-type delta (PTPRD) gene as a therapeutic strategy.
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.
Research at Technical University of Munich found that diabetes causes loss of small blood vessels around the heart, affecting cardiac muscle. Genetic therapy promoting pericyte growth may offer a remedy, providing new perspectives for treating patients.
A new study from MU researchers has identified a protein that can be targeted to decrease post-heart attack injury and prevent heart failure in a mouse model. The research used ultrasound technology to deliver a protein inhibitor directly to the hearts of mice, resulting in a 72% decrease in heart damage compared to control groups.
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.
A new study found that genotyping and personalized warfarin dosing can lower the risk of certain adverse events, such as bleeding and blood clots. The Genetic InFormatics Trial showed a 27% reduction in adverse events with genotype-guided dosing compared to clinically guided dosing.
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.
Researchers at the National Eye Institute used CRISPR to rescue photoreceptors in mice, preserving daylight and color vision. The approach could lead to novel therapies for preventing vision loss from human diseases such as retinitis pigmentosa.
A team of researchers has identified a gene that causes Opitz C syndrome in the only patient diagnosed with this ultra-rare disorder in Catalonia. The study used DNA massive sequencing techniques to identify the altered genes in each patient, providing new insights into the genetic basis of this disease.
A study published in the Public Library of Science Pathogens found that Zika virus targets neuronal, lymph, joint, muscle and genital/urinary/reproductive tissues, persisting for at least 35 days. The research provides insights into the growth and distribution of the virus in human tissues.
Scientists construct five new artificial yeast chromosomes, representing over one-third of yeast's entire genome, paving the way for building the first fully synthetic complex organism. The successful assembly demonstrates genetic plasticity and potential applications in gene therapy, biofuel production, and medicine.
Researchers from the University of Würzburg discovered four variants of the GLRB gene associated with anxiety and panic disorders, triggering an
A study by Boston University School of Medicine researchers has identified common and unique patterns of protein synthesis associated with increased longevity. This discovery could lead to the development of new therapeutic targets for human diseases such as type 2 diabetes, cancer, and cardiovascular disease.
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.
New research concludes that AAV vectors used in gene therapy do not increase the risk of cancer and may even have anti-tumorigenic properties. The study's authors review biomedical evidence showing that AAV2 can negatively impact other viruses associated with malignancy.
Scientists have engineered the smallest CRISPR-Cas9 system to date, capable of delivering gene editing to muscles and eyes via AAV. This technology targets age-related macular degeneration (AMD) and other 'undruggable' genes, offering a promising therapeutic tool.
Researchers at MDI Biological Laboratory identified a molecular mechanism governing the life-prolonging effects of dietary restriction, a process that occurs in all tested animals. The study raises hope for therapies that prolong healthy years without extreme diet restrictions.
A novel gene therapy technique, called charge-altering releasable transporters (CARTs), has been developed to deliver messenger RNA (mRNA) into cells. This method overcomes the challenge of delivering mRNA through cell membranes and shows promise for treating diseases by inserting therapeutic proteins.
Drs. Marco Gallo and Greg Delgoffe receive ACGT Young Investigator Grants to study glioblastoma and metastatic melanoma, focusing on gene therapy and T cell metabolism to boost immune responses against cancer.
Researchers have used gene therapy to treat Fabry disease, a rare inherited enzyme deficiency that can damage major organs and shorten lifespan. The treatment involves transplanted altered stem cells with copies of the fully functional gene responsible for the missing enzyme.
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.
Researchers at Salk Institute use mRNA therapy to deliver instructions for clotting protein, achieving normal clotting and minimal immune response in mice. The therapy shows potential as a cost-effective and safer alternative to existing treatments for hemophilia B and other genetic diseases.
Lipid nanoparticles (SLNs) have shown increased effectiveness in delivering nucleic acids, with advantages including protection against degradation and boost to transfection process. Researchers are exploring their application in treating various diseases, including degenerative disorders of the retina, infectious diseases, and cancer.
Researchers validated 70 genes associated with congenital heart disease in fruit flies, including those modifying histone structure. This approach may enable precise gene-editing techniques for tailored treatments.
Researchers have developed a new technique using DNA 'barcodes' to rapidly screen nanoparticles for therapeutic delivery. The method allows hundreds of different types of nanoparticles to be tested simultaneously in just a handful of animals.
A new assay has been developed to detect genetic mutations in tumors, showing high sensitivity and specificity in detecting actionable genomic variants. The NCI-MATCH trial aims to evaluate tumor biopsy specimens from 6,000 patients using this technology.
Researchers have successfully restored a high level of hearing in deaf mice using an improved gene therapy vector, which also improves balance function. The technology has the potential to benefit patients with Usher syndrome, who suffer from profound hearing loss and balance problems.
Researchers at Queen Mary University London have discovered a genetic defect causing kidney and adrenal disease in patients from around the world. The study found that defects in the SGPL1 gene can lead to progressive diseases with neurological involvement.
A new method called synthetic essentiality has been found to identify therapeutic targets in cancers lacking specific key tumor suppressor genes. By analyzing gene deletion patterns, researchers identified CHD1 as a potential treatment site for prostate and breast cancers with PTEN gene loss.
Dr. Stanley T. Crooke presents a detailed look at the mechanisms underlying antisense drug activity, including prehybridization, hybridization, and post-hybridization phases. He highlights his group's contributions to characterizing ASO therapeutics' design, targeting, and mechanism of action.
A recent study has identified 83 DNA variants that modulate human height, with some affecting it by more than 2 cm. The discovery is significant for understanding the genetic basis of complex diseases such as diabetes and schizophrenia, and may lead to the development of new therapeutic strategies.
Researchers developed a new gene-editing method called homology-independent targeted integration (HITI) that efficiently inserts DNA into genes in dividing and non-dividing cells of living rats. The technique uses the CRISPR-Cas9 tool to cut DNA at a specific location, followed by the NHEJ repair pathway for insertion.
Researchers developed a mini-intronic plasmid (MIP) system that can enhance transgene expression by up to 40-100-fold when used with adeno-associated viral (AAV) vectors. This could reduce the cost of gene therapy and lessen immune responses.
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.
Targeting therapeutic genes to the lungs offers potential for managing serious lung diseases that do not respond to other treatments. Preclinical and human clinical studies have targeted various genetic diseases, including cystic fibrosis, asthma, and lung cancer.
Researchers found that inhibiting PLSCR1 controls the infected cell's antiviral response, providing long-term protection from immune attack and excessive inflammation. This discovery holds promise for virally delivered treatments, inflammatory conditions, autoimmune disorders, and neurodegenerative diseases.
Scientists identified collateral lethal vulnerability in pancreatic cancers that can be targeted pharmacologically in certain patient populations. ME3 inhibitors may provide an effective therapy for many cancer patients.
Researchers found a significant excess of mutations in five genes previously implicated only in rare forms of epilepsy in individuals with common forms of the disorder. The study identified these genes as contributing to epilepsy risk in approximately 8 percent of people with familial non-acquired focal epilepsy.
Researchers at Northwestern University have discovered a genetic driver of mixed lineage leukemia, a rare and deadly form of childhood leukemia. They identified a targeted molecular therapy that halts the proliferation of leukemic cells by stabilizing the wild-type MLL protein, which drives cancer.
Scientists at Roswell Park Comprehensive Cancer Center have identified gatekeeper genes that allow prostate cancer to progress and resist treatment. The study highlights opportunities to prevent or reverse this process, offering new insights into lineage plasticity and its application in other types of cancers.
Katherine High discusses gene therapy progress for hemophilia and inherited retinal disorders, highlighting key scientific, regulatory, and commercial challenges. Her leadership in the field has been critical to its current success.
A new research from Michigan Medicine supports combining immune checkpoint blockade with gene therapy to fight gliomas. The approach showed stronger results when used in combination with either depletion of immunosuppressive cells or immune checkpoint blockade.
This study investigates molecular changes in the OTOF gene in patients with auditory neuropathy and identifies the c.35delG mutation in the GJB2 gene. Cochlear implants show excellent outcomes, with improved speech development and language acquisition in patients with severe/profound hearing loss.
Gene therapy for liver disease is advancing with the help of adeno-associated viral (AAV) vectors, offering new therapeutic possibilities. The article discusses preclinical and clinical studies that have led to a better understanding of immune responses to AAV gene therapy.
Researchers evaluated two adeno-associated virus vectors in non-human primates to assess gene expression efficiency and immunogenicity. The results provide insight into the safety and effectiveness of these gene therapy approaches before initiating clinical trials in humans.
Researchers have identified rare genetic variants linked to observable physiological characteristics in over 50,000 individuals. This approach has the potential to inform healthcare and prevent undiagnosed single-gene disorders like familial hypercholesterolemia.
Researchers used CRISPR-Cas9 gene-editing technology to identify three promising new targets for HIV infection. The study screened human genes essential for HIV replication but not for cellular survival, and identified five genes with potential therapeutic applications.
Researchers identified four new markers of primary sclerosing cholangitis risk on the human genome, bringing the total number of known predisposing locations to 20. The study also shed light on the genetic relationship between primary sclerosing cholangitis and inflammatory bowel disease.
Researchers identified five genes that, when inactivated, protected cells from HIV infection without affecting cellular survival. The genes target human proteins essential for HIV replication and viral entry into CD4 T cells.
Researchers at the University of Rochester Medical Center have identified a potential new treatment approach for lysosomal storage disorders, which cause disruptions in cellular functioning. The study found that repurposed drugs can overcome toxic build-up in affected cells, improving survival rates and quality of life.