Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
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.
Researchers at UTMB have identified distinct subtypes of asthma using protein profiling, which could lead to tailored treatments. The study found four different asthma subtypes, including one associated with severe and treatment-resistant disease, offering new hope for more effective asthma therapies.
Researchers compared DNA of patients with reduced MS symptoms to those experiencing relapses, identifying key genetic markers associated with treatment response. The study's findings may help predict which treatments will work best for individual patients.
Researchers at the University of Rochester Medical Center have developed a new graft technique using freeze-dried tendon grafts loaded with gene therapy, showing significantly fewer adhesions than traditional autografts. The study demonstrates promising results in restoring range of motion and accelerating healing after hard-to-treat i...
University of Florida researchers have successfully triggered insulin production in diabetic mice by injecting them with the naturally occurring protein Pdx1. The study found that daily injections of Pdx1 normalized blood glucose levels within two weeks, promoting regeneration of insulin-producing cells in the pancreas.
China's health biotech industry boasts daring medical science innovation and stunning breakthroughs, including the world's first commercialized gene therapy product. However, firms face an uphill battle in attracting high-risk venture capital needed for innovative projects due to uncertain financial systems and export restrictions.
Researchers used rats bred to be heavy drinkers and administered an anti-Aldh2 antisense gene, reducing consumption by half for a month. The study suggests gene therapy could be a promising approach for treating alcoholism, particularly in individuals with genetic mutations protecting them from the risk of developing the condition.
Researchers identified brain regions that fire up more when people make impulsive decisions, a key finding linking thinking patterns to addiction. The study suggests raising dopamine levels may be an effective treatment for addiction, offering new approach to therapy and potential subtypes of alcoholics.
Researchers have alleviated various abnormalities associated with fragile X syndrome by reducing the activity of a specific gene. The findings suggest that this approach may lead to treatment for the disorder, which affects brain structure and function, as well as causing mental retardation, epilepsy, and abnormal body growth.
A research team has uncovered the molecular mechanism by which osteocrin controls bone growth, a discovery that may have important implications for people suffering from bone diseases affecting skeletal growth. Osteocrin was found to locally control the bone's supply of a hormone known as CNP, regulating bone growth.
A new study published by Mayo Clinic researchers found that Herceptin helps women with HER2+ type of breast cancer independent of whether they have extra copies of chromosome 17. The analysis showed significant benefits for patients with polysomy 17, including a disease-free survival rate of 89% after three years of treatment.
Researchers found that alcohol activates a stress-linked pathway in neurons to release key genes that can influence the health and activity of brain cells. The study sheds light on how this process occurs and may help scientists understand alcohol-linked disorders such as chronic alcoholism and fetal alcohol syndrome.
A study found that antidepressant non-responders with depression show a significantly better response to lithium augmentation if they carry at least one C-allele, suggesting a potential genetic predictor for treatment response. The GSK3B gene variation may be a key molecular switch related to the clinical effectiveness of lithium.
Dr. Bridges' group successfully demonstrates marker genes can be efficiently inserted into heart muscle cells in large animals using a novel surgical procedure and specially designed hardware. The approach has the potential to develop new treatments for various heart muscle diseases causing heart failure, with preliminary success showi...
Researchers block single gene NF-êB to reverse aging in mouse skin, demonstrating that aging is a result of an active genetic program. The study sets the stage for future genetic age-intervention therapies.
Researchers are conducting a phase 1 clinical trial using gene therapy to treat LCA2, aiming to restore vision in individuals with inherited blindness. The treatment involves injecting trillions of replacement genes into the retina, using an adeno-associated virus to deliver the therapeutic gene.
A recent study using PET scans shows that an experimental gene therapy can normalize brain function in Parkinson's patients. The treatment was found to alter brain activity in a favorable way, primarily affecting motor networks.
Researchers found that gene signatures can predict which chemotherapy regimens are most effective for breast cancer patients. The study showed a significant improvement in response rates when using gene signatures to select treatments, suggesting a potential breakthrough in personalized medicine.
Researchers found that antibody responses to viruses like measles and mumps remain protective for several decades, even for life. This means people may not need booster shots as frequently as previously thought, potentially reducing unnecessary vaccinations.
A Buck Institute study found that lithium increases lifespan in nematode worms by reducing the activity of a gene that modulates chromosome structure. The research highlights the potential for using C. elegans as a model organism to study drug toxicity and genetic impacts of compounds.
Gene therapy administered intravenously protected healthy tissues in mice during whole-body irradiation, with 90% survival rate at 30 days. The therapy offered no harm to systemic function and could potentially provide wide-reaching public protection in radiation emergencies.
Researchers at Cedars-Sinai Medical Center have developed a novel gene delivery system that can sustain therapeutic expression in the central nervous system for up to 1 year. This innovative approach uses an adenoviral vector called HC-Adv, which is invisible to the immune system and renders previously used vectors ineffective.
Researchers have identified two genes, ARTS1 and IL23R, that increase the risk of developing ankylosing spondylitis, a common disease causing back pain and progressive stiffness. A potential treatment for Crohn's disease may also be applied to this disease, providing a major step towards being able to treat ankylosing spondylitis.
Researchers at Cincinnati Children's Hospital Medical Center found that Hypoplastic Left Heart Syndrome (HLHS) has high heritability and is likely caused almost entirely by genetic effects. Families with a child with HLHS carry a significant recurrence risk of HLHS or related heart defects.
Researchers at Children's Hospital of Philadelphia successfully delivered beneficial gene to entire mouse brain with one injection, correcting diseased areas. The technique has potential for treating rare genetic neurological disorders like Tay-Sachs disease and Sly syndrome.
Researchers at the University of Missouri-Columbia have identified 400 genes that can be manipulated to make chemotherapy drug cisplatin more effective. These genes, involved in sphingolipid metabolism, are linked to whether a tumor cell survives or dies after treatment.
New nonviral gene delivery systems utilizing transposons provide a safer alternative to viruses, with potential benefits for treating fatal conditions such as cancer. The technology has shown promise in achieving stable expression of genes in animal cells and minimizing insertional mutagenesis risk.
Researchers identified 17 genes linked to high-risk multiple myeloma, which can predict poor prognosis and guide therapy. The activity of these genes could help categorize patients' risk early, enabling personalized treatment plans.
A recent study found that a specific genetic variant affects smoking cessation treatment outcomes, with 45% of individuals benefiting from bupropion treatment and maintaining abstinence longer compared to those without the variant.
Gene therapy holds great promise but faces safety concerns due to virus-based delivery methods. MIT researchers have created biodegradable polymers that can deliver genes safely and effectively, showing promise for ovarian cancer treatment and other applications.
Researchers at Rensselaer Polytechnic Institute are developing a personal device to measure daily light intake and activity, predicting optimal timing for light therapy to synchronize the circadian clock. The device aims to alleviate psychosocial stress and sleep-related ailments caused by inadequate or irregular light exposure.
Researchers develop bacteria to deliver anti-cancer treatments by targeting oxygen-starved areas of tumors. The treatment shows effective results in experimental animals, offering a promising alternative to traditional cancer therapies.
Researchers at UT Southwestern Medical Center have discovered a single gene that controls fat formation in various organisms, including humans. The 'adipose' gene is believed to be a high-level master switch that determines whether the body accumulates or burns fat.
Researchers developed a genetic dosing model to predict optimal warfarin dose for patients undergoing orthopedic surgery. The model combines genotype information with clinical factors to estimate therapeutic dosage.
A research team has identified a genetic deficiency that causes severe blindness, specifically Leber Congenital Amaurosis (LCA). The discovery of the LCA5 gene and its protein lebercilin provides new opportunities for gene therapy, which could lead to the treatment of this disease in humans.
Researchers at Virginia Commonwealth University identified two key proteins, KLF2 and EKLF, that regulate overlapping groups of genes during red blood cell development. The study may lead to future gene therapies for sickle cell anemia and beta-thalassemia.
Researchers developed a new vaccine strategy that uses a protein-free M. Tuberculosis strain to activate immune cells, providing increased protection against TB. A study found that vaccination with this strain provided better protection than the existing BCG vaccine.
A study published in the Journal of Clinical Investigation reveals that variants of the TCF7L2 gene increase susceptibility to type 2 diabetes by affecting insulin secretion in pancreatic islet cells. The findings provide a mechanism for genetic predisposition and suggest potential therapeutic targets for new treatments.
Researchers identified over 50 genetic abnormalities in sporadic ALS, providing expanded opportunities for developing treatments. The study's findings also uncovered genes controlling nerve adhesion, offering a major new avenue for ALS research.
Researchers have developed a biodegradable nanoparticle delivery system that uses magnetic forces to target specific cells in the body. The system has shown promise in reducing cell proliferation and delivering anti-growth genes to stents, which could help maintain blood flow.
Researchers at Duke University Medical Center discovered a novel gene associated with increased susceptibility to multiple sclerosis, which increases risk by 30%. The gene variant affects the function of interleukin-7 receptor alpha, guiding immune system cell development and growth.
Researchers created a tunable genetic switch to regulate gene expression, overcoming limitations of existing technologies. The switch enables precise control over gene function, offering potential applications in basic research, gene therapy, and cell and gene therapy.
Researchers at WashU Medicine have identified hundreds of DNA elements that control when and where genes linked to blindness are turned on. These new elements can be used as switches to activate blindness therapies, offering hope for a potential cure for inherited blindness.
Researchers at Children's Hospital of Philadelphia identified a gene variant associated with an increased risk of Crohn's disease in children. The study found that 64% of children with the disease had a specific variant form of the ATG16L1 gene, compared to 52% of healthy children.
Scientists at UCSD School of Medicine have found that 'junk' DNA sequences may serve as punctuation marks to organize functional domains within the genome. This discovery could lead to breakthroughs in gene therapy by understanding how genomic material contributes to the regulation of genes.
A new study by Ohio State University researchers links high ERG gene activity to a more lethal subtype of acute myeloid leukemia (AML). Patients with high ERG expression are almost six times more likely to relapse or die within five years, highlighting the need for more intense therapy.
Researchers at the University of Texas M. D. Anderson Cancer Center have developed a gene therapy that selectively targets and eliminates pancreatic cancer cells, inhibiting metastasis and prolonging survival with minimal toxicity. The therapy, called VISA-BikDD, has shown significant promise in preclinical trials, with treated mice su...
Researchers at Penn University have successfully restored brain function to blind animals using gene therapy, showing promise for treating genetic retinal disease. The treatment, which involves introducing a working copy of the RPE65 gene into the retina, also revived responses in the visual cortex of the canine brain.
A novel gene therapy technique has been found safe and effective in reducing worsening symptoms of Parkinson's disease in advanced patients. The treatment, which involves injecting genes into one side of the brain to restore a critical neurotransmitter called GABA, showed a 27% improvement in symptoms among participants.
A new gene therapy clinical trial has reported promising results in improving motor function for patients with Parkinson's disease. The study found significant improvements in both the off-state and on-medication phases, with some patients showing impressive gains of up to 65 percent.
A study published in The Lancet found that gene therapy can safely alleviate symptoms of Parkinson's disease, with significant improvements seen in movement and motor scores. The treatment involves injecting genetic information directly into brain cells, eliminating the need for indwelling hardware associated with deep-brain stimulation.
A Phase 1 study using an adeno-associated virus vector to deliver an inhibitory gene to the subthalamic nucleus demonstrated a lack of adverse events and statistically significant improvements in clinical symptoms and brain metabolism. The treated side showed notable improvement, with sustained benefits observed over 12 months.
Researchers discovered previously unknown exons outside annotated genes in human proteins, suggesting complex gene regulation and increasing the risk of disease-causing mutations. This finding has significant implications for medicine, requiring extra caution when studying genetic phenotypes.
Scientists have discovered a way to transfer genes into diseased tissue of the human inner ear, aiming to restore hearing. The breakthrough could lead to the development of gene therapy compounds that produce new hair cells and restore hearing function in humans.
Researchers develop new therapy to re-activate silenced genes, potentially blocking cell death in stroke patients. The therapy targets histone deacetylase enzymes, which can modulate gene expression and produce neuroprotective proteins.
Researchers delivered gene therapy containing nerve growth factors to rats with erectile dysfunction, resulting in significant recovery of intracavernous pressure and systemic arterial pressure. The study suggests a long-term treatment option for erectile dysfunction without chronic drug administration.
Researchers developed a smaller gene therapy vector to deliver a radioprotective enzyme systemically, sparing healthy tissue from radiation damage. The minicircle plasmid conferred undiminished radioprotection to cells, suggesting improved treatment outcomes for cancer patients.
An international team of researchers has been awarded £2 million to study the genetic causes of dwarfism and develop future treatments. The EuroGrow project aims to investigate the most common bone disorders that lead to short stature, with a focus on achondroplasia, pseudoachondroplasia, and spondyloepiphyseal dysplasia congenita.
Researchers successfully used gene therapy to block pain response in an animal model of neuropathic pain. The study involved delivering a genetically engineered herpes simplex virus to rats with the goal of activating the glycine receptor, which alleviated pain response. This breakthrough suggests that gene therapy may be effective for...
Researchers used a recombinant adeno-associated virus to study genomic instability in sporadic cancers, identifying breakage-prone palindromes that can contribute to cancer and aging. The discovery provides an opportunity to study these sequences on a whole genome scale.