Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
The Cancer Genome Atlas identifies new genetic mutations, including NF1, ERBB2, and PIK3R1, and core pathways disrupted in glioblastoma. The study provides an unprecedented overview of the genomic landscape of this deadly cancer.
A study has identified a new gene that may play a key role in the development of pediatric-onset inflammatory bowel disease. The gene, TNFRSF6B, is involved in regulating inflammation and its activity has been linked to the severity of the disease.
A new study suggests that short interfering RNAs (siRNAs) may exhibit non-specific therapeutic effects related to immune stimulation. The researchers used a mouse model of influenza and demonstrated that the anti-viral activity of siRNA is mainly due to non-specific stimulation of the immune system.
A study has identified the first gene associated with severe dry macular degeneration, a condition that can lead to blindness. The researchers found that individuals with a specific genetic variant may be at risk if treated with an experimental therapy called RNA interference.
Researchers have discovered a genetic mutation that offers protection against age-related macular degeneration (AMD), a leading cause of blindness in adults over 50. The study identifies a functional link between toll-like receptor 3 (TLR3) and geographic atrophy, a type of dry AMD.
A new study found that an experimental therapy for age-related macular degeneration may have adverse effects on patients with a certain genetic mutation. The therapy uses RNA interference to silence disease-causing genes, but this approach may not be suitable for everyone due to individual genetic differences.
University of Alabama at Birmingham researchers have developed a gene therapy virus to target ovarian cancer cells. The adenovirus-based approach has shown anti-tumor effects that appear safe for most patients, according to Drs. David T. Curiel and Ronald Alvarez.
Researchers developed PTC124 to bypass premature 'stop' signals in CFTR genes, allowing the protein-making machinery to function normally. The phase II trial showed improvements in chloride ion transport and lung respiratory function, offering a potential treatment for cystic fibrosis.
A novel mutation in the nephronophthisis 4 (NPHP4) gene has been identified as a potential cause of cone-rod dystrophy in standard wire-haired dachshunds. This discovery raises hopes for developing treatments for humans with similar eye disorders.
Researchers explore new treatments for psoriasis, revealing a potential therapeutic option in AEB071, while cautioning further clinical trials are needed. Additionally, studies uncover the reasons behind gene therapy complications in SCID patients, shedding light on insertional oncogenesis and acquired somatic mutations.
A team of researchers has developed a powerful tool to induce specific genomic modifications in many types of cells, including plants and humans. The new method, known as OPEN, enables academic researchers to rapidly create high-quality enzymes that can target and manipulate DNA sequences for repair or inactivation.
A multi-institutional team has developed a powerful tool for genomic research and medicine, generating synthetic enzymes that can target specific DNA sequences for inactivation or repair. This new method, called OPEN, enables customized zinc-finger nucleases with high efficiency in many cell types.
Researchers identified six genes associated with autism by analyzing large Middle Eastern families where both parents shared a recent ancestor. The study suggests that these genes affect learning and memory, and may be treatable through gene therapy or enriched learning environments.
A study of Middle Eastern families with a high incidence of autism identified six new genes involved in the brain's ability to form new connections. The findings suggest that autism may stem from disruptions in experience-dependent learning, which is essential for learning and memory.
Scientists at UGA create novel synthetic gene vectors that package DNA into nanostructures for efficient gene delivery without triggering immune responses. The study has implications for cancer treatment and vaccine development.
A small study of 18 patients with severe Marfan syndrome found that losartan slowed aortic enlargement by nearly half, offering a promising new treatment option. The study's findings have led to the approval of a large-scale clinical trial for losartan in Marfan syndrome patients.
The Indiana University School of Medicine will be home to the nation's sole National Gene Vector Biorepository and Coordinating Center for gene therapy research. The center will help researchers share information and substances to promote discoveries and patient safety.
Researchers developed a therapeutic system targeting HIF-1 activated cancer cells, showing success in laboratory tests. The system uses a reporter gene to detect and treat hypoxic cancer cells that are resistant to traditional treatments.
A new gene therapy has helped pet dogs with cancer live longer by increasing muscle strength and correcting common complications. The treatment has been shown to improve quality of life and survival rates, with 54% of dogs responding to the therapy and surviving 84% longer.
Research by NARSAD scientists provides clues on how genes impair attention, memory, and perception in psychiatric illnesses like schizophrenia, bipolar disorder, and depression. The study focuses on specific genes and family traits that contribute to the susceptibility and pathology of these diseases.
Researchers identified genetic variants that influence treatment outcome for smoking cessation treatments, including nicotine replacement therapy (NRT) and bupropion. These variants may help tailor treatment to individual smokers, increasing the likelihood of success.
Researchers have developed artificial viruses that can transport genes and drugs into cancer cells, eliminating immune responses and potential side effects. The viruses are self-assembled using a ribbon-like protein structure, allowing for precise control over their size and shape.
Researchers at the Burnham Institute have demonstrated successful enzyme replacement therapy to prevent hypophosphatasia in mice, paving the way for future clinical trials. The treatment maintains healthy bone mineral density and preserves skeletal structures, offering new hope for patients with this genetic disorder.
A University of Rochester team has identified approximately 100 genes that cooperate to cause malignant cell transformation, providing a potential roadmap for next-generation cancer therapies. These genes, known as CRGs, encode proteins that regulate cell signaling and metabolism.
New data reveals genetic variation in AKT1 affects cognition and grey-matter volume in the frontostriatal region, a brain area dysfunctional in individuals with schizophrenia. This association may increase the risk of developing the condition.
Researchers investigated using gene therapy to inhibit NHE1 in human gastric carcinoma cells, leading to apoptosis of tumor cells. The study found that inhibition of NHE1 reduced malignant phenotypes and restrained tumor cell survival.
Researchers at the University of Florida have developed a new version of the adeno-associated virus used in gene therapy that works more efficiently than current vectors. By replacing an amino acid on the surface of the virus, they were able to improve its ability to deliver genes into cells and reduce the risk of triggering an immune ...
Researchers presented findings on Maxi-K gene therapy, showing it to be safe and restoring erectile function in men with moderate to severe ED. The treatment has been shown to enhance sexual behavior, including increases in partial and full erections and a two-fold increase in erection duration.
Researchers have successfully treated late infantile neuronal ceroid lipofuscinosis (LINCL) with gene therapy, slowing disease progression in children. The treatment, which replaces the faulty CLN2 gene, demonstrated significant improvements over an 18-month period.
A clinical trial found gene therapy to be safe and effective at slowing disease progression in Batten disease, a rare genetic disorder. Neurological function was assessed using a rating scale throughout an 18-month follow-up period.
Researchers discovered glypican-3 loss induces overgrowth through Sonic Hedgehog, a growth factor stimulating cancer growth in rare disorder SGBS. This finding opens doors for novel treatments to inhibit overgrowth activity benefiting SGBS patients and GPC3-related cancer patients.
The American Society for Neural Therapy and Repair (ASNTR) has awarded neuroscientist Paul M. Carvey for his groundbreaking research on Parkinson's Disease, while recognizing patient advocate Joan Samuelson for her tireless advocacy efforts.
A new study by UC Davis researchers provides evidence that methods using human bone marrow-derived stem cells to deliver gene therapy do not cause the development of tumors or leukemia. The study tested the safety of gene transfer into bone marrow stem cells in over 600 mice, with none developing leukemia or solid tumors.
A genetic breakthrough explains dangerously high blood glucose levels in healthy individuals by identifying a DNA sequence that controls variability. This discovery holds out hope for 'personalized medicine' and may lead to customized treatment based on an individual's unique genetic structure.
Researchers have successfully used gene therapy to restore vision in three young adults with a rare form of congenital blindness. The study showed significant improvements in visual acuity and sensitivity to light, setting the stage for further studies.
Researchers developed a breakthrough in gene therapy for cancer using microscopic magnets to target tumours, increasing successful cell invasion. The 'magnetic targeting' method uses nanomagnets to attract cells carrying anti-cancer genes, effectively overcoming the problem of insufficient delivery.
Researchers have discovered a new vector, AAV5, that can carry large genes linked to inherited blindness, paving the way for potential gene therapy treatments. This breakthrough enables the delivery of corrected faulty genes into cells where they are required.
New data shows that vectors derived from AAV5 can accommodate large genes, including those missing in a mouse model of recessive Stargardt disease. This breakthrough has implications for treating individuals with the disease, which is characterized by progressive loss of sight.
Cardiology researchers at The Children's Hospital of Philadelphia have developed a synthetic material that can bind to various gene therapy vectors, allowing controlled local release of therapeutic genes. In an animal study, the new formulation showed significant reduction in restenosis, a hazardous narrowing of blood vessels.
Researchers have identified two potential biomarkers that could help doctors monitor the effectiveness of treatment with sunitinib or bevacizumab for kidney and non-small cell lung cancer. A study found that circulating endothelial progenitor cells (ccEPCs) increase in patients who respond to therapy, while those who do not respond hav...
Researchers have identified a crucial role for antibodies in protecting against nontyphoidal strains of Salmonella bacteria, which causes fatal infections in African children. Meanwhile, a study on HOXB4 gene therapy found that early precursors can cause leukemia in large animals, highlighting the need for extreme caution in human trials.
Researchers at St. Jude Children's Research Hospital are investigating two areas of molecular science that hold promise for improving the survival and quality of life of ALL patients, including adolescents and adults. Gene-expression profiling and pharmacogenetics may help identify targets for future therapy and reduce toxicity.
Researchers at Northwestern University discovered a counterintuitive approach to gene therapy that targets the removal of genes to restore cellular function. This method has implications for medical research and optimizing metabolic processes used in biofuel production.
Researchers made a significant breakthrough in understanding the physics of translocation, showing that memory effects in polymeric molecules dominate their behavior. This discovery has major implications for drug delivery and gene therapy, as well as single-molecule characterization techniques.
Researchers have successfully treated mice with a gene responsible for making follistatin, a protein that inhibits myostatin, leading to long-term improvements in muscle mass and strength. The therapy has potential for older patients with Duchenne muscular dystrophy, who often lose treatment options once muscles degenerate.
Researchers identified a genetic variation in the VKORC1 gene that determines a patient's initial sensitivity to warfarin treatment. The study found that only variations of VKORC1 predict response within the first week of therapy, which could lead to better warfarin dosing and increased safety.
Researchers developed gene therapy approach that attracts and 'trains' immune system cells to destroy deadly brain cancer cells, promoting long-term immunity and restoring normal brain function. The therapy shows promise as a potential treatment for glioblastoma multiforme, the most common and deadly type of brain cancer.
Researchers at University College London have identified a combination of genes in Asian monkeys that protects against retroviruses, including HIV. By mimicking these genes in humans, scientists hope to develop a novel gene therapy treatment for HIV/AIDS.
Researchers at McGill University have discovered a genetic process that boosts an organism's natural anti-virus defences by knocking out genes repressing interferon production in mice. This breakthrough could lead to the development of new anti-viral therapies in humans, providing a potential solution to combat viral pandemics.
A recent MUHC study reveals that cystic fibrosis-related bone problems are an additional pathology stemming from the Cftr gene mutation, not a side effect of treatment. The study found that mice with the mutation have lower bone mineral density and mass than control mice, without pancreatic insufficiency or steroid treatment.
Researchers found that STAT3 has tumor-promoting and tumor-suppressing effects depending on the genetic profile of glioblastoma tumors. This discovery highlights the need for effective therapies tailored to individual glioblastoma tumors.
Researchers at Henry Ford Hospital are conducting a phase III clinical trial to determine the effectiveness of gene therapy in combination with radiation therapy for patients with intermediate risk prostate cancer. The trial aims to improve patient outcomes by delaying hormone therapy, which can have severe side effects.
Researchers have found that tattooing is a more effective way of delivering DNA vaccines than intramuscular injection, producing stronger humoral and cellular immune responses. This method could have a role in routine vaccination of cattle or delivering therapeutic vaccines to humans.
Scientists use gene therapy to breed goats producing therapeutic proteins in their milk, reducing drug development time and cost. This breakthrough increases drug availability for those who cannot afford treatments.
RNA interference represents an innovative strategy for silencing specific genes associated with disease processes, and a series of review articles will focus on its potential therapeutic applications. The technology has been approved for at least six clinical trials and is considered to be here to stay.
A series of commentaries in Human Gene Therapy explores key controversies surrounding patient recruitment and trial management in gene therapy trials. The authors discuss the challenges of patients comprehending lengthy and technical study descriptions and consent forms, and propose innovative strategies for improving informed consent.
Researchers at Mount Sinai School of Medicine have discovered a novel targeted gene therapy that simulates the pain-killing effect of opiate drugs, providing relief for more than three months after a single injection. The technique targets selectively the pain gate, avoiding unwanted side effects associated with opioid painkillers.
A landmark genetic study identified multiple genes linked to systemic lupus erythematosus (SLE), a debilitating autoimmune disease affecting joints, kidneys, heart, lungs, brain and blood. The findings will ultimately lead to new therapies and earlier diagnosis, with 13 strong candidates supported by data that are 99% accurate.
Researchers at Thomas Jefferson University have discovered that USP22 is crucial for controlling large-scale changes in gene expression in cancer cells. The study reveals that USP22 is necessary for the global changes in gene expression patterns that occur in these cells, making it a potential target for new anti-cancer drugs.
Researchers at Scripps Research Institute identified a nonsense mutation in the Coronin-1A gene that suppresses lupus development in mice. The study suggests that this mutation and other disease-suppressing genes may play a crucial role in modulating autoimmunity.