Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
A new study has identified genes more frequently altered in prostate tumors from men of African ancestry compared to other racial groups. The research found no significant differences in frequencies of mutations in genes important for current prostate cancer therapies, but suggests that precision medicine approaches can be effective if...
Researchers successfully deliver gene therapy using nanoparticles to inhibit abnormal blood vessel growth in rats and mice eyes, providing evidence for treating wet age-related macular degeneration and inherited retinal diseases.
A recent report of two children's deaths in a gene therapy trial for X-linked myotubular myopathy has sparked concerns over the safety of gene therapy vectors. The editorial emphasizes the need for iterative development and cooperation among scientists to ensure safe and effective treatments for rare genetic diseases.
An international team identified an error in the cytochrome P450 oxidoreductase (POR) gene as the cause of aromatase deficiency, a rare metabolic disorder. This discovery enables precise therapy and prevents side effects associated with steroid supplementation.
The SARS-CoV-2 virus activates the aryl hydrocarbon receptors (AhRs) through the IDO1-kynurenine-AhR signaling pathway, leading to Systemic AhR Activation Syndrome (SAAS). This triggers a cytokine storm causing inflammation, thromboembolism, and fibrosis. Researchers propose therapies targeting downregulation of AhRs and IDO1 genes to ...
Researchers have developed a reagent for selective and safe coating of the liver sinusoidal walls to control clearance of gene therapy drugs. The coating agent improved gene transfer efficacy by 2-4 times to the myocardium and skeletal muscles, and 10 times to colorectal cancer, reducing medical costs and adverse effects.
Researchers at Kobe University have developed an exon-skipping therapy using antisense oligonucleotides to treat Alport Syndrome, a genetic kidney disease. The treatment was found to be effective in reducing urinary protein levels and suppressing kidney failure in model mice with severe mutations.
Researchers at the University of Iowa have developed a gene therapy approach to convert lab mice into models of COVID-19. This tool allows researchers to rapidly test treatments and vaccines, including poly I:C, convalescent plasma, and remdesivir, which prevented weight loss and lung disease in infected mice.
A study published in JAMA Neurology reports successful delivery of micro-dystrophin to patients with Duchenne muscular dystrophy (DMD), resulting in functional improvements. The therapy showed robust gene expression and localization, as well as improved muscle function measured by North Star Ambulatory Assessment scores.
Scientists have developed a new gene therapy approach that offers tremendous promise for treating an eye disease that leads to blindness. The collaborative team used a modified virus to deliver a normal functioning copy of the RP2 gene into 'mini retinas' containing the defective version, resulting in significant improvement.
A Phase 3 study found lumasiran to be effective in reducing 24hr urinary oxalate excretion by 65.4% and normalizing oxalate levels in patients with PH1. The treatment also showed an encouraging safety profile, with mild injection-site reactions being the most common adverse event.
A research team from Genethon has successfully inhibited the immune response induced by AAV antibodies, paving the way for repeated administration of gene therapy treatments. This breakthrough could enable treatment of rare genetic diseases and improve patient outcomes.
Italian scientists have identified common genetic susceptibility genes linked to COVID-19 outcomes, including severe pneumonia. The study's findings suggest that these genes may determine disease progression and could aid in the development of new treatments.
A Phase Ib/II trial found that adding idh1 inhibitor ivosidenib to either venetoclax or azacitidine resulted in a high complete remission rate of 78% overall, with 100% for treatment-naïve patients. The combination may ultimately lead to a new therapeutic regimen and tailored treatment options based on molecular profiles.
Nanoscope Technologies has developed a novel, non-viral gene delivery approach using low-power near-infrared lasers to target the retina. The method sensitizes cells to ambient light, improving visual response and offering a unique therapy option for dry-AMD patients.
Research identified potentially treatable genetic mutations in prostate cancer patients who did not respond to PSMA-targeted therapy. Targeted next-generation gene sequencing found mutations in six out of seven patients, including alterations in DNA damage-repair genes such as TP53, ATM, and CHEK2.
Researchers discover Foxg1's role in controlling brain electrical activity, shedding light on rare genetic diseases like Rett Syndrome and West Syndrome. Understanding this mechanism is crucial for developing future therapeutic interventions.
A study in mice suggests gene therapy can build significant muscle mass quickly and reduce the severity of osteoarthritis, even without exercise. The therapy also prevented obesity and improved cardiovascular health.
Researchers identified two previously overlooked genes as potential new therapeutic targets for cancer treatment and male infertility. RMPs play a key role in the RNA modification process, adding or removing chemical groups to the sequence, and altering the original code copied from DNA.
University of Virginia researchers are pioneering a new approach using focused ultrasound to deliver treatments directly into the brain. The technology, which can selectively open the blood-brain barrier, has shown promising results in treating conditions like Alzheimer's, epilepsy, and brain tumors.
A study identified a rare genetic variant in the ANGPTL7 gene that lowers intraocular pressure and significantly reduces the risk of glaucoma. The findings open up new therapeutic possibilities for treating this eye disease.
Researchers identified two distinct protein shapes associated with Parkinson's disease, multiple system atrophy, and dementia with Lewy bodies. The discovery may lead to improved diagnosis and treatment of these age-related conditions.
A new study has discovered a link between gene defects and eczema, wheeze, and nasal blockage among babies as young as six months. The research suggests that treatment targeted towards children carrying these genetic defects started soon after birth could improve their lives.
The Phase III EMBRACA trial found talazoparib improved patient-reported quality-of-life measures and had a tolerable safety profile in patients with metastatic HER2-negative breast cancer and BRCA1/2 mutations. However, the study did not demonstrate a statistically significant overall survival benefit due to subsequent treatments.
Researchers found that FDA-approved drugs Betaxolol and Pravastatin can increase muscle strength in mouse models of DMD by stimulating utrophin production. This approach could lead to novel therapies for Duchenne muscular dystrophy, potentially offering advantages over traditional gene replacement methods.
Researchers found that a specific gene variant, klotho, can protect against Alzheimer's disease in people with the ApoE4 high-risk gene variant. This finding could help identify individuals at lower risk and inform clinical trial participants and treatments.
A clinical trial using genetic testing to guide antiplatelet medication found a 34% reduction in serious cardiovascular events at one year, as well as a significant decrease in the number of events per patient. This finding suggests that genetically guided therapy may be beneficial for patients with specific genetic variants.
The National Gene Vector Biorepository (NGVB) provides valuable resources to gene therapy investigators, including 93 unique reagents and a searchable database of animal safety studies. These resources aim to decrease compliance risks, address clinical trial funding periods, and reduce costs.
Researchers at University of California San Diego School of Medicine identified a novel way to treat Danon disease using gene therapy, adding a specially designed gene that restores LAMP2 function. The treatment improved cardiac and liver function in mice with the disease, offering a new approach beyond heart transplants.
Using high-throughput screening of adeno-associated viral (AAV) vector capsid libraries, researchers identified functional and efficient AAV variants after only one round of selection. Infection with a high multiplicity of infection (MOI) was found to be preferable to infection with a low MOI, reducing variation between screens.
Researchers at Boston Children's Hospital have successfully used gene therapy to reverse cardiac dysfunction and scarring in two mouse models of Barth syndrome. The treatment, which replaced the mutated TAZ gene, prevented progressive cardiomyopathy and restored heart function in both whole-body and heart-only TAZ deletion mice.
A novel gene therapy has been developed to regenerate functional new neurons in mouse models of Huntington's Disease, offering a potential treatment for the condition. The therapy uses NeuroD1-based gene therapies to convert brain internal glial cells into functional new neurons.
Scientists from the University of Illinois at Urbana-Champaign developed a new CRISPR gene-editing methodology that inactivated a key gene responsible for ALS, slowing disease progression and improving muscle function. The treatment also increased overall survival in mice with aggressive forms of ALS.
Duchenne muscular dystrophy researchers have identified a potential new method for therapies, using small molecules to increase utrophin protein production. This approach bypasses the immune system's challenges and may provide functional characteristics similar to dystrophin.
The Clinic for Special Children's SMA Prevention Readiness program successfully identified 318 carriers and 9 affected couples, treating 3 infants with gene therapy. The program's cascade testing approach was effective in detecting genetic risk, leveraging funding from biotech company AveXis.
Researchers at Karolinska Institutet have discovered a gene, Synaptotagmin 13, that protects against motor neuron degeneration in ALS and SMA. Gene therapy was shown to increase life expectancy in animal models.
Researchers delivered an ALDH2 gene to mice with the deficiency, using a virus-mediated approach. The treated mice showed no signs of the acute abnormalities or chronic disorders normally associated with ethanol exposure in ALDH2 deficiency.
A clinical research program at Columbia University Irving Medical Center will treat eight more ALS patients with FUS mutations using the investigational therapy jacifusen. The program aims to expand access to treatments for rare ALS subtypes associated with gene mutations.
A team of scientists from Purdue University and international research institutions developed a new method for identifying nonviral vectors in gene therapies. This approach uses big data, patent, and clinical data mining to uncover emerging trends in the field. The study aims to guide future developments in gene therapy.
Scientists have developed a simple and precise cancer therapy approach based on selective electrochemical generation of H2 in tumor regions. The H2-ECT method enables large-scale tumor therapy with ideal selectivity and targeting to kill tumors with minimal damage to normal tissue.
Researchers at St. Jude Children's Research Hospital are exploring genetically engineered immune cells to attack pediatric sarcoma using a new ACGT grant. The approach aims to target not only cancer cells but also blood vessels supporting tumor growth, offering a promising new treatment for children with solid tumors.
Researchers have developed a new single AAV gene therapy platform that can treat almost any mutation, showing improved vision in blind mice and paving the way for clinical trials by 2025.
A team of UPenn researchers is developing a new CAR T-cell gene therapy treatment for advanced metastatic prostate cancer, with the goal of overcoming resistance to current treatments. The ACGT-funded study aims to re-engineer T-cells to induce safe, long-term remission in patients.
Researchers analyzed ovarian cells from young and old non-human primates to understand ovarian aging. They identified genes that could be used as biomarkers and point to therapeutic targets for diagnosing and treating female infertility and age-associated ovarian diseases.
Researchers at the University of Missouri have made a groundbreaking discovery by identifying the genetic mutation responsible for Chédiak-Higashi syndrome in domestic cats. This finding has significant implications for treating inherited diseases and preventing their transmission to future generations.
A gene therapy trial conducted in US and UK patients with X-linked Chronic Septic Granulomatosis, a rare genetic disease causing severe infections, has shown success. Six of nine patients are free of treatment complications, offering hope for sustainable treatment.
Researchers identified specific regions of chromosome 21 causing memory and decision-making problems in mice with Down syndrome. The study found that different gene groups on mouse chromosomes 16, 10, and 17 contribute to cognitive issues.
Researchers at UCLA and international partners have successfully treated nine patients with X-linked chronic granulomatous disease (X-CGD) using a stem cell gene therapy. Six patients are now in remission and have stopped other treatments, with no complications reported.
Researchers at Technical University of Munich used CRISPR-Cas9 gene scissors to correct the mutated dystrophin gene in living pigs, improving muscle function and life expectancy. The therapy has shown promising results in a clinically relevant large animal model, mirroring Duchenne muscular dystrophy in humans.
A team at Massachusetts Eye and Ear has identified GPR108, a G protein-coupled receptor, as a molecular 'lock' necessary for most adeno-associated virus (AAV) vectors to gain access to cells. This discovery may enable scientists to better explain, predict, and ultimately direct AAV gene transfers to specific tissues.
A recent study published in PNAS reveals that extracellular matrix protein-1 (ECM1) plays a crucial role in the pathogenesis of ulcerative colitis, a type of inflammatory bowel disease. The researchers found that ECM1 is highly expressed in macrophages and regulates their polarization through the GM-CSF/STAT5 signaling pathway.
A study found that an overactive immune system gene causes schizophrenia-like changes in mice, including neural and behavioral aspects of the disease. The researchers suggest that this gene may be contributing to the development of schizophrenia through mis-regulated synaptic pruning.
Researchers have identified a second gene, BSND, which determines the severity of kidney disease in patients with Joubert syndrome. The discovery has significant implications for diagnosis and treatment of genetic kidney diseases, offering hope for personalized therapies to reduce disease severity.
Researchers develop biodegradable nanoparticles that target and kill pediatric brain tumor cells in mice, surviving 20-63% longer than untreated mice. The treatment uses a combination of the suicide gene and ganciclovir, showing promise for new therapies targeting these deadly brain malignancies.
Researchers at Michigan Technological University have developed a single-particle method using atomic force microscopy to determine virus isoelectric points, improving the accuracy of characterization and paving the way for more efficient vaccine production and gene therapy manufacturing.
A study found that APLN overexpression correlates with worsened prognosis in ovarian cancer patients treated with bevacizumab. The researchers also identified a distinct gene signature associated with resistance development, paving the way for new treatment strategies.
Researchers at the Mayo Clinic have developed a single-dose gene therapy to treat cocaine addiction. The therapy uses a recombinant adeno-associated viral vector to deliver a gene that metabolizes cocaine into harmless byproducts.
A breakthrough gene therapy treatment has been shown to offer long-term benefits for people with Haemophilia A, transforming the lives of 13 UK patients. The treatment resulted in a substantial fall in bleeding rates three years after treatment, with none requiring regular factor VIII injections.
Researchers developed an adeno-associated virus vector to deliver anti-pTau antibodies directly into the hippocampus of mouse models with CTE, reducing pTau levels across the CNS. The study suggests this strategy could be effective in humans and may offer a new treatment option for CTE.
Researchers at Mayo Clinic tested a new injection method for delivering gene therapy vectors directly into the kidney, outperforming traditional intravenous injections. The study found that direct injections were superior to intravenous injections and had fewer off-target effects.