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 uses gene transfer to block the expression of an enzyme that breaks down alcohol, leading to increased acetaldehyde levels and potential treatment for alcoholism. The approach has strong proof-of-concept data and bodes well for future development of a genetic therapy.
Researchers identified macrophage migration inhibitory factor (MIF) and its related protein D-dopachrome tautomerase as closely related cytokines associated with progressive MS. A genetic test could identify patients at risk of developing the disease, accelerating drug development for precision medicine.
Researchers at Brigham and Women's Hospital discovered a rare genetic mutation in the ALK gene that causes peritoneal mesothelioma in young women with no history of asbestos or radiation therapy. Targeted therapy using FDA-approved drugs may be effective in treating these patients.
Clinical evidence supports the use of mesenchymal stem cell transplantation to treat Crohn's disease-related fistulas, offering a safe and effective approach. The unique advantages of MSCs include ease of collection, low immunogenicity, and immunoregulatory activity.
A genetic mutation in the CLPX gene has been identified as a potential cause of erythropoietic protoporphyria (EPP), a form of porphyria that may have inspired vampire folklore. The discovery highlights the complex genetic network underlying heme metabolism and holds promise for future therapies.
Neuroscientists at the University of Nevada, Reno identified a genetic mechanism in fruit flies that disrupts brain pathways connecting the left and right hemispheres, separately linked to autism. The study suggests that the human gene PRRG4 may play a role in regulating nerve fiber guidance and synapse formation.
A new clinical investigation is testing whether genetic screening can identify patients with a specific mutation who are the best candidates for deep brain stimulation surgery. The study aims to determine how this genetic information can inform surgical decisions and potentially improve outcomes for Parkinson's disease patients.
The FDA has approved Kymriah, a CAR T gene therapy drug, for pediatric and young adult patients with ALL, marking a major milestone in cancer treatment. The therapy showed an exceptional 83 percent success remission rate in clinical trials.
Researchers at Fred Hutchinson Cancer Center developed a nanoparticle delivery system to extend the therapeutic potential of messenger RNA, delivering gene changes to targeted cells. The 'hit-and-run' genetic programming approach can be easily scaled up for various disease applications.
Researchers used gene therapy with LeXis, a 'junk DNA' suppressor of cholesterol, to reduce plaque in mice with familial hypercholesterolemia. The treatment lowered cholesterol and blockages in arteries, and appeared to reduce fat build-up in liver cells.
Scientists explore gene therapy for injured peripheral nerves, aiming to improve functional recovery after microsurgical repair. The approach involves modifying a directional cue for nerve cells to promote fast regeneration and functional recovery.
A team led by Dr. Rena D'Souza aims to identify the relationship between PAX9 and WNT pathways in permanent tooth formation. The goal is to develop new therapies that reestablish normal interaction between genes and pathways to restore tooth development.
Researchers hope IBM's Watson will help people with cancer by providing detailed information on genetic mutations and targeted therapies. With Watson, pathologists can analyze multiple genes simultaneously, including those that are not currently tested, to provide more personalized care to patients.
Researchers at the University of Chicago have developed a novel gene therapy approach that uses skin transplants to treat type-2 diabetes and obesity. The method uses CRISPR technology to deliver a hormone that stimulates insulin production, reducing blood glucose levels and promoting weight loss.
Researchers developed a gene therapy using microdystrophin to treat Duchenne muscular dystrophy in dogs. The treatment restored muscle function and stabilized clinical symptoms for over 2 years. This breakthrough could lead to a new treatment for children with the disease.
The Massachusetts General Hospital team, led by J. Keith Joung, is developing more sensitive methods of measuring and controlling on-target gene-editing activity and detecting off-target mutations induced by CRISPR-Cas9 technology.
A comprehensive study published in Nature Communications reveals that lean body mass is highly heritable and can be attributed to specific genes. The research may lead to the development of future therapies to prevent the loss of lean mass and associated disabilities such as sarcopenia.
The Phase 3 clinical trial data showed statistically significant and clinically meaningful improvements in functional vision and visual field in participants with RPE65-mediated inherited retinal disease. Participants demonstrated marked gains in full-field light sensitivity and peripheral vision.
The FDA advisory committee voted unanimously to recommend approval of Novartis' CAR-T therapy Tisagenlecleucel, demonstrating impressive results in hard-to-treat leukemia patients. ACGT's funding played a crucial role in advancing the pioneering treatment.
A proof-of-concept study demonstrates the potential of nanoparticles in delivering molecules that target specific genetic markers in glioblastoma brain tumors. The therapy stops tumor growth and extends survival when administered continuously through an implanted drug infusion pump.
Researchers have developed novel secreted reporter proteins that can be detected for several months after infusion into the liver, demonstrating potential for monitoring therapeutic gene delivery and ongoing expression. The findings show promise for scaling up this method to humans.
Researchers have successfully delivered a gene therapy to the brains of mice with mucopolysaccharidosis type 1, preventing neurologic deficits. The noninvasive intranasal approach shows promise for treating genetic disorders affecting the brain.
Researchers at Wayne State University are investigating how genetic factors impact the benefits of exercise, using a fruit fly model to identify novel targets for therapeutic pharmaceuticals. The study aims to understand how exercise adaptations can be induced in patients who cannot perform regular physical activity.
A study has identified a common gene mutation linked to retinitis pigmentosa in Hispanic families from the Southwestern US, with over 70 genes now known to cause the disease. The researchers found that a dominant mutation in the arrestin-1 gene is responsible for 36% of cases, offering hope for future treatments and therapies.
A study has characterized the mouse genome, revealing new insights into rare diseases and accelerating the development of new treatments. The research identified 360 new disease models and 28,406 new descriptions of genes' effects on mouse biology and disease.
A new study published in Cell suggests that virtually any gene can influence disease, with peripheral genes having tiny but significant effects. The 'omnigenic model' proposes a more nuanced understanding of genetic variation and its role in disease.
Researchers found that artificial viruses can infect brain cells and surrounding tissues beyond the injection site, influencing the immune response. The study's findings could improve the selection of suitable viral 'gene transporters' for custom therapies, offering a glimmer of hope for patients with Alzheimer's and Parkinson's.
Researchers have discovered a solution to evade the immune system, allowing more patients to participate in gene therapy clinical trials. The altered virus can slip past neutralizing antibodies, making it possible for up to 70% of patients previously ineligible to join trials.
Researchers at German Cancer Research Center (DKFZ) have discovered mysterious gene transcripts that arise in treated cancer cells. These transcripts, originating from ancient viral elements, may be used as biomarkers to monitor the effectiveness of epigenetic therapy.
A new study found that oral prednisone reduced immune responses to AAV-based gene delivery, resulting in a 60% decrease in cytotoxic T cell infiltrates. The treatment also increased PD-L2 levels, which induce programmed T-cell death, suggesting a potential therapeutic benefit for gene therapy.
Researchers conducted a study assessing stem cell transplantation for patients with a rare 'gain of function' STAT1 gene mutation. The treatment showed encouraging results, with five patients being completely cured and disease-free, but the overall success rate was disappointing at 40%. Experts propose adjusting treatment parameters, s...
A new gene therapy has been shown to preserve muscle strength and neurologic function in dogs with myotubular myopathy, a type of muscular dystrophy. The therapy, which involves replacing the mutated MTM1 gene, has provided long-lasting benefits over 4 years.
Recent clinical trials using different gene transfer approaches have shown acceptable risk and potential for treating heart failure with reduced ejection fraction. Despite mixed results in terms of efficacy, the data support further clinical development of this therapeutic strategy.
A French study of 1,944 patients with advanced cancer found that comprehensive genomic profiling can guide treatment choices and lead to higher survival rates. The study's results suggest that routine genomic testing may not benefit all patients but offers hope for personalized medicine.
A study found that combining therapies targeting polyADP ribose polymerase (PARP) and mitogen-activated protein kinase (MEK) inhibitors showed promise in treating RAS-mutant cancers. The combination therapy was effective in multiple tumor models, regardless of mutations in tumor suppressor genes.
Researchers in China have made a breakthrough in developing a new approach to treat premature ovarian failure using female germline stem cells. The study found that mice with transplanted egg-making stem cells produced healthy pups without genetic malfunctions, opening up avenues for investigating human oogenesis in vitro.
Researchers have developed a novel gene therapy method that utilizes microbubbles and ultrasound to promote bone growth and healing in nonunion fractures. The technique, which involves injecting genetic material for a bone growth factor into the affected area, has been shown to completely heal broken bones in pigs within eight weeks.
Researchers have shown that an experimental gene therapy is safe and may be effective in preserving vision in people with wet age-related macular degeneration. The treatment involves injecting a virus containing a therapeutic gene into the eye, which produces a protein to prevent abnormal blood vessel growth.
Researchers have discovered a more favorable virus species for delivering genes, as its surface structure avoids liver toxicity. This could enable the use of viruses in gene therapies and vaccine development.
Researchers at Case Western Reserve University developed a gene-carrying nanoparticle that delivers healthy RPE65 genes to photoreceptor cells, preventing vision loss in mice with Leber congenital amaurosis. The therapy's success indicates promise for treating other inherited visual disorders.
The study discovered genetically coupled promoters in viral-host networks, presenting an additional layer of regulation between virus and host. The findings reveal a mechanism for synchronizing gene expression in synthetic gene circuitry and provide novel design principles to reverse-engineer viral circuitry.
A new review discusses how gene therapy delivered via adeno-associated viruses (AAV) can trigger an immune response in T cells, leading to activation and exhaustion. The review highlights the variability in individual patients' immune responses to AAV vectors and potential strategies to suppress these reactions.
A growing number of preclinical studies suggest that rAAV-mediated gene therapy may cause insertional mutagenesis and increase the risk of hepatocellular carcinoma. Research emphasizes the need for careful reconsideration of potential genotoxicity risks in human clinical applications.
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.
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.
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.
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 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.
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.