Researchers have developed a medicated foam that can deliver expensive gene therapies to cells, showing promise for treating cancers and autoimmune diseases. The foam outperformed liquid formulations in laboratory studies and showed no significant side effects when injected into mice.
A novel gene cluster expression analysis was introduced to potentially expand treatments for more lung cancer patients. The study found that a gene cluster expression index can classify cancers with dramatically different recurrence risks, with higher risk groups suitable for targeted therapy or immunotherapy.
Researchers at Fred Hutch Cancer Center have made progress in laboratory studies using gene editing to eliminate herpes virus infections. The experimental therapy eliminated 90% of HSV-1 after facial infection and 97% after genital infection, reducing viral shedding.
Researchers at UW–Madison developed a method using short electric pulses to increase gene therapy material uptake in liver cells, reducing dosage needed. The technique could help treat genetic diseases like cystic fibrosis and sickle-cell disease.
A clinical trial found that video-only hand therapy is non-inferior to in-person therapy after thumb CMC arthroplasty, offering a more patient-driven approach with reduced travel burden. Video-administered therapy provided similar outcomes and significantly reduced travel distance and time for patients.
Columbia University has received a $15 million grant to develop tailor-made gene-based therapies for nine people with rare forms of amyotrophic lateral sclerosis (ALS). The Silence ALS program will use antisense oligonucleotide (ASO) technology to silence genes that produce toxic proteins in motor neurons. The project aims to create pe...
A clinical trial at NIH demonstrated that experimental gene therapy slowed loss of motor function in patients with giant axonal neuropathy, a fatal childhood disease. The treatment also showed signs of regeneration in sensory nerves, offering new hope for those affected.
A Kyoto University research group developed RENGE, a computational model to estimate gene regulatory networks in multicellular organisms. The method measures time-series gene expression and uses the proprietary model to infer regulatory dynamics.
Recent clinical trials highlight a new challenge in AAV gene transfer: complement system activation, which may contribute to adverse events and immune priming. The article provides a comprehensive review of the complement cascade and its interactions with AAV vectors.
Researchers found that manual therapy provided a moderate and statistically significant reduction in disability among adults with sacroiliac joint pain syndrome. However, the efficacy of manual therapy for pain related to this syndrome is unclear at this time.
A breakthrough gene-editing therapy has been successfully treated over ten patients with hereditary angioedema, reducing symptoms by up to 95% and potentially providing a permanent cure. The therapy targets the KLKB1 gene responsible for producing plasma prekallikrein.
Researchers have found that a gene-based therapy targeting plakophilin-2 can interrupt the progression of arrhythmogenic right ventricular cardiomyopathy, a rare inherited disorder. The treatment reduced episodes of arrhythmia and slowed the deterioration of the heart's walls in mice.
A novel gene therapy has been shown to restore hearing function in six children with DFNB9, a form of autosomal recessive deafness. The treatment, which uses an adeno-associated virus carrying a version of the human OTOF gene, demonstrated significant improvements in speech perception and ability to conduct normal conversation.
Researchers identified key characteristics associated with improved CAR T outcomes in large B cell lymphoma, including a B-cell gene expression signature and high CD19 protein expression. Patients who received axicabtagene ciloleucel had better event-free survival compared to those receiving standard therapy.
Researchers at Baylor College of Medicine have developed a technology to effectively regulate gene expression, promising a safer approach to gene therapy. The system uses small molecules to interact with RNA, allowing for precise control over protein production and therapeutic window maintenance.
Researchers at UCL Queen Square Institute of Neurology have developed a new gene therapy that significantly reduces seizures in mice with focal cortical dysplasia. The treatment, which involves the overexpression of a potassium channel, has shown an average reduction of 87% in seizures without affecting the mouse's memory or behavior.
Gene therapy treatments can prompt an adverse immune reaction when the body mistakenly perceives the treatment as a viral threat. Researchers have identified a critical pathway in the liver that triggers this response, but also found a way to block it, paving the way for safer and more precise gene therapy options.
Researchers discovered a link between gene therapy and the accumulation of stem cells with genetic mutations, which can lead to accelerated growth and increased blood cancer risk. The study suggests that younger patients may be safer candidates for gene therapy due to fewer genetic mutations.
A new review article highlights significant milestones in gene therapy for rare diseases, improving efficacy and safety. However, challenges remain to make this transformative approach widely available.
Researchers at the University of Texas Health Science Center discovered a gene therapy approach that can restore motor functions in mice with Contactin-Associated Protein 1 (Cntnap1) mutations. The study found that turning on the normal gene earlier improves the rescue outcome, and the next phase is to test this approach in humans.
Researchers identified the integration site pattern of lentiviral gene therapies in patient cells, shedding light on treatment safety and efficacy. The study found that integration near nuclear pores is associated with improved safety and effectiveness, avoiding oncogene regions where earlier technologies failed.
A new study found that delivering gene therapy to the cerebrospinal fluid directly corrects gene mutations causing CLN2 disease, showing potential therapeutic effects. In nonhuman primates, TPP-1 activity increased significantly after treatment, providing hope for families affected by this fatal childhood disorder.
Researchers at Rice University have developed a non-invasive gene delivery technique using ultrasound to efficiently deliver clinically used gene therapy vectors throughout the brain. The study, published in Gene Therapy, shows that opening more sites within targeted regions improves gene delivery efficiency.
A new clinical trial published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for sickle cell disease. The therapy was shown to decrease vaso-occlusive events and increase production of fetal hemoglobin, providing relief from the painful condition.
Researchers at UCL and NIHR Great Ormond Street Hospital Biomedical Research Centre have developed a gene therapy that significantly reduces hearing loss in mice with Norrie disease. The treatment, which restores the missing norrin protein, has shown improved blood vessel structure and protected sensory hairs in the inner ear.
Researchers have engineered an AAV vector to target brain vascular pericytes and smooth muscle cells, potentially treating conditions like Alzheimer's disease and Parkinson's. This breakthrough could revolutionize gene therapy by delivering genes directly to the brain's blood vessels.
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.
A novel gene delivery procedure was successfully applied to baboons, achieving therapeutic levels of human factor IX gene expression lasting up to 200 days. The study demonstrated the efficacy of repeated hydrodynamic gene delivery into the same liver lobes, with no significant adverse events observed.
A team of scientists has developed an ex vivo gene therapy approach to treat obesity and diabetes in mice by implanting cells expressing the CPT1AM protein, which plays a key role in metabolic diseases. The study shows promising results, reducing weight, fatty liver, cholesterol, and glucose levels in obese mice.
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.
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.
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.
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.
Researchers at Michigan State University have developed a gene therapy that successfully treats a form of progressive retinal atrophy in dogs with an inherited eye disease. The therapy is now being prepared for human clinical trials to treat retinitis pigmentosa, a rare genetic disorder causing vision loss.
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.
A phase 2 clinical trial found improved neurocognitive outcomes and comparable survival rates for children with craniopharyngioma treated with proton therapy compared to photon therapy. The study highlights the therapeutic advantage of proton therapy in sparing normal brain tissue and reducing collateral effects.
Scientists developed a new RNA-based toolkit that can regulate gene expression, potentially improving regenerative medicine, gene therapy, and biotechnology. The technology uses small molecules to control the activity of synthetic RNA, allowing for precise control over gene expression.
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...
Dr. Crystal Mackall, a leader in advancing cell and gene therapies for children's cancers, is the newest recipient of the Edward Netter Leadership Award. She has led numerous clinical trials treating children with sarcomas and brain cancers.
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 examine the effects of gender-affirming androgen therapy on breast tissue in transgender men with single-cell resolution. Gene expression is silenced, and male gene programs are activated. This work has implications for the treatment of breast cancer across all populations.
A new study led by OHSU researchers reveals that gene editing technology in human embryos can lead to unintended changes in the genome and may not accurately reflect gene edits. The study highlights the need for caution when using genetically edited embryos to establish pregnancies.
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 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.
A new UCLA-led study found that many patients do not receive sufficient rehabilitation therapy after a stroke, which can lead to long-term disability. Hispanic patients were particularly affected, receiving lower amounts of physical and occupational therapy.
A new approach to gene therapy for inherited blindness uses lipid nanoparticles to deliver mRNA inside the eye, targeting light-sensitive cells and creating proteins that edit vision-harming gene mutations. The technology has shown promising results in animal studies, including mice and nonhuman primates.
A new treatment approach using surgery or radiation therapy has been shown to slow cancer progression in patients with solitary prostate cancer metastases. The study found that this approach, called metastasis-directed therapy (MDT), can delay the need for hormone-blocking treatments and improve survival rates.
A new study presents a closed-loop gene therapy approach that targets only overactive neurons, reducing spontaneous seizures in mice. The approach uses the Fos gene to control the Kcna1 gene, promoting reduced neuronal excitability and offering a persistent antiepileptic effect.
Researchers at the University of Pennsylvania School of Medicine have developed a gene therapy that rapidly restores night vision in adults with LCA, a common form of congenital blindness. The treatment has been shown to improve rod-based visual functions and correct severe visual deficits in patients.
Researchers developed new educational materials to help individuals with sickle cell disease and their support networks learn about gene therapy clinical trials, benefits, risks, and treatment options. The project aims to facilitate engagement between the community and those conducting relevant clinical trials.
Researchers have developed a gene therapy that rescues cilia defects in retinal cells affected by Leber congenital amaurosis, a disease causing blindness in early childhood. The therapy restores opsin protein concentration in photoreceptor outer segments, suggesting functional NPHP5 may stabilize the primary cilium gate.
Researchers from Trinity College Dublin have developed a new gene therapy approach targeting mitochondrial function to treat dry AMD, a debilitating eye disease. The therapy has shown benefit in multiple models of the condition, offering hope for a potential treatment that could help millions globally.
A new type of adeno-associated virus (AAV) gene therapy candidate, FLT180a, has been shown to reduce bleeding risk in patients with haemophilia B. The treatment led to sustained production of FIX protein from the liver in nine out of ten patients, eliminating the need for regular replacement therapy.
A large cohort study found that hormone replacement therapy for breast cancer survivors is not associated with an increased risk of recurrence. The study included over 8,400 Danish postmenopausal women and found no link between vaginal estrogen or menopausal hormone therapy and breast cancer reoccurrence.
Researchers aim to develop a gene therapy that could suppress HIV replication and eliminate the need for life-long antiretroviral therapy. The study uses an AAV vector to produce leronlimab, a monoclonal antibody blocking HIV entry into immune cells.
The North American Menopause Society has released its 2022 Hormone Therapy Position Statement, reaffirming the effectiveness of hormone therapy in treating hot flashes and other menopause symptoms. The updated statement also highlights its role in preventing bone loss and fractures.
Researchers have developed a gene therapy that appears to correct a rare creatine deficiency disorder by increasing creatine levels and reducing toxic guanidinoacetic acid. This could hold promise for treating the disorder, which commonly results in intellectual disabilities and seizures.
A proof-of-concept study suggests that postnatal gene therapy may prevent or reverse deleterious effects of Pitt-Hopkins syndrome, a rare genetic disorder. The treatment restored normal activity of the deficient gene, preventing anxiety-like behavior and memory problems in an animal model.
A gene therapy trial has shown significant reduction in infections and improvement in eczema symptoms in children with Wiskott-Aldrich syndrome (WAS). The treatment, using lentiviral gene therapy, offers a safe alternative to bone marrow transplantation for patients without suitable donors.