Articles tagged with Gene Therapy
Researchers are developing a new gene therapy to treat CTLA-4 insufficiency, a life-limiting inherited immune disorder. The treatment involves replacing a faulty gene in a patient's own immune cells using CRISPR/Cas9 technology.
Researchers developed a novel gene therapy platform that successfully restored muscle function in preclinical models of Duchenne muscular dystrophy by delivering full-length mRNA of the DMD gene via engineered extracellular vesicles. The treatment showed improved muscle strength, endurance, and function without serious side effects.
Researchers identified a promising new strategy for reversing autism-related brain deficits by targeting a specific glycine transporter. The therapy restored NMDA receptor function in mouse models and human brain organoids, improving behavioral abnormalities such as social interaction and repetitive behaviors.
Researchers develop a gene therapy approach to restore normal nerve signaling and reduce muscle spasticity in spinal cord injury patients. The treatment targets GABA signaling and has shown significant improvements in reducing spasticity and restoring reflex function.
A new study by the American Society of Hematology finds that stem cell transplantation is the most cost-effective option for long-term care of sickle cell disease in adults. Gene therapy, while promising, comes with an astronomical cost and requires a significant price reduction to be competitive.
A new technology allows for the efficient insertion of large DNA segments, enabling a 'chapter rewrite' in the genome. This method avoids double-strand breaks and can correct hundreds of mutations simultaneously.
The researchers will investigate novel therapies to protect the aging brain from neurodegenerative diseases by eliminating RNA pollution. They will map out signatures of RNA pollution across over 200 cell lines and patient biofluids to understand its effects.
A new study improves gene editing efficiency by identifying key genes in human cells that drive particle assembly. By disabling a single gene, researchers boosted production of potent delivery vehicles. The discovery has broad implications for the widespread application of gene editing technologies.
A study coordinated by the University of Trento has identified a gene therapy for individuals with cystic fibrosis caused by a specific mutation, offering new hope for those currently dependent on drugs. The therapy uses advanced gene editing technology to correct the DNA mutation responsible for the disease.
A new international study shows that gene therapy targeting the OTOF gene successfully restored hearing in most participants, with significant improvements in speech perception and language skills. The treatment remained safe and effective for up to 2.5 years, offering new hope for individuals with inherited deafness.
Dr. Stuart Orkin has been awarded the Breakthrough Prize in Life Sciences for discovering genetic mechanisms underlying hemoglobin production and developing a therapy using CRISPR/Cas9 technology. The therapy, Casgevy, treats patients with sickle-cell anemia and beta-thalassemia, debilitating blood disorders affecting millions worldwide.
Scientists corrected a genetic disease of the liver in mouse models and human patient cells using gene editing, building a foundation for a potential new therapy for Zellweger spectrum disorder. The breakthrough restored function of the liver and peroxisomes, reducing toxic buildup.
Researchers have discovered a smaller CRISPR enzyme, Al3Cas12f, that can efficiently edit genes in human cells. The enzyme's unique structure allows it to form a stable connection with DNA, making it a promising candidate for therapeutic genome editing.
Researchers used base editing to correct the SCN8A gene mutation responsible for severe inherited epilepsy. The approach successfully eliminated or reduced seizures and improved brain function in lab mice, offering new hope for treating genetic epilepsies.
Researchers have created genetically modified marmosets with a knocked-out OTOF gene, replicating key characteristics of human deafness. The animals developed normally but were deaf from birth, offering a crucial tool for developing new therapies.
Researchers at University of Michigan Engineering and Michigan Medicine used protein nanoparticles to genetically modify several types of human cells, including liver cancer and immune cells. The goal is to develop a safer method for delivering gene therapies without using modified viruses.
The FDA has approved a gene therapy for severe leukocyte adhesion deficiency-I, a rare genetic condition that affects one in a million children globally. The therapy, Kresladi, has been shown to significantly reduce severe infections and improve immune function in treated patients.
Researchers at USC will develop an AI-driven framework to strengthen evidence generation for gene and cell therapies, helping bring promising treatments closer to patients. The project aims to better understand how specific therapy features relate to patient outcomes.
A new RNA therapy has been developed to enhance the heart's own ability to protect and repair itself after a heart attack. The therapy, which involves injecting particles into the arm, significantly reduced scarring and improved heart function in lab experiments, offering a potential breakthrough for heart patients.
A new approach, called INSTALL, enables non-toxic DNA integration in multiple human cell types and successfully inserts large genetic payloads in mice, offering a promising solution for genetic therapies. The study's findings have the potential to broaden the applicability of genome editing therapies.
Genethon's GNT0004 gene therapy shows long-term efficacy in patients with Duchenne muscular dystrophy, maintaining clinical efficacy and safety at two years. The trial included 72 boys aged 6-10 with retained walking ability, treated with GNT0004 at a therapeutic dose.
The company's ATA-200 gene therapy has shown safety, pharmacodynamics, and efficacy results in the first patients treated, offering hope for children with LGMD-R5. The therapy delivers a normal copy of the γ-sarcoglycan gene and has been awarded Orphan Drug Designation in the US and Europe.
Phase 1/2a clinical trials demonstrate significant seizure reduction and improvement in symptoms of Dravet syndrome, a genetic disorder affecting cognitive function, motor skills, and behavior. The treatment, zorevunersen, targets the underlying cause of the disease by enhancing the normal SCN1A gene.
A gene therapy platform successfully mapped the living brain noninvasively, using engineered proteins to track gene expression in different brain regions. This technology has the potential to reveal critical information about cellular activity and neurological disease progression.
Engineers have refined a technology to edit individual genetic base pairs, reducing unintended edits and increasing safety for potential treatments. The new base editors could lead to better outcomes for some cystic fibrosis patients and more accurate models for drug testing.
Researchers at Tokyo Metropolitan University have created a neutral molecule that can carry DNA into biological cells using a process called annealing. This breakthrough promises more effective therapies by reducing inflammation and improving delivery efficiency.
A new Immunology Center will accelerate discoveries in muscle immunology and immune responses to gene therapies. Klaudia Kuranda brings expertise in immunology, onco-immunology, and leadership experience to the center.
Rice bioengineer Jerzy Szablowski has been awarded a Sloan Research Fellowship for his innovative work on noninvasive methods to communicate with the brain. He aims to develop versatile platform technologies to obtain new types of research data and treat multiple diseases.
Harlequin ichthyosis is caused by ABCA12 mutations leading to defective lipid transport and loss of skin barrier function. Management includes neonatal care, systemic retinoids, daily emollients, and keratolytics, with improved survival rates and quality of life.
Researchers in Lund have presented a new model for cooperation that will shorten lead times and reduce costs. The Cell and Gene Therapy Navigator tool helps identify imbalances and future bottlenecks in projects.
A bibliometric analysis of global advances in cell and gene therapy reveals uneven progress, with US and China leading the field. Japan's contributions are significant but lack qualitative influence.
Aviv Regev, a pioneering computational biologist, will deliver a keynote address on tissue stem cells at the ISSCR 2026 Annual Meeting. Her work has transformed our understanding of cell and tissue function in health and disease.
David J. Segal has been appointed as the chair of UC Davis Department of Biochemistry and Molecular Medicine, known for his groundbreaking research in gene-editing technologies. He is developing targeted molecular tools to treat rare genetic disorders, including Angelman syndrome and neurofibromatosis type 1.
Researchers at the University of California - San Diego have developed a new method to improve gene therapy by increasing the efficacy of gene delivery while minimizing harmful side effects. The new workflow allows for increased control of nuclear DNA delivery, with greater than tenfold increase in nuclear DNA delivery observed.
Researchers developed AAVLINK, harnessing Cre/lox-mediated intermolecular DNA recombination to enable in vivo reassembly of large genes. The method achieves high-efficiency full-length gene reconstitution and significantly improves therapeutic outcomes in animal models.
A new study by McGill University researchers offers a potential solution for infant hydrocephalus, a life-threatening condition that affects 1 in 1,000 newborns. RNA therapy has been shown to prevent the condition in mice models, with a promising impact on treating genetically caused hydrocephalus.
The ISSCR is hosting a summit on access and affordability in cell and gene therapies to explore pricing, manufacturing, regulation, and reimbursement strategies. Experts will examine key barriers and emerging solutions across the access landscape.
Researchers at University of California San Diego discover gene therapy restoring connexin-43 improves heart function and extends survival in several forms of arrhythmogenic cardiomyopathy. The approach may have broader therapeutic potential across multiple genetic forms of the disease, addressing a critical unmet need.
The collaboration aims to deliver life-saving treatments at a fraction of their current market cost, using Caring Cross's decentralized manufacturing model. Clinical data demonstrated robust increases in fetal hemoglobin with sustained mitigation of SCD disease manifestations.
Researchers at Rice University are exploring biological systems-inspired delivery vehicles to target specific tissues in living organisms, aiming to improve the efficiency of gene-based therapies. The project focuses on optimizing combinations of surface molecules to enable precise and efficient delivery of large DNA payloads.
Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells. Genethon's gene therapy approach has shown preclinical efficacy in animal studies, correcting glycogen accumulation and improving cardiac hypertrophy and muscular dysfunction.
The CRISPR-Cas3 system has been shown to induce reliable and extensive deletions of the TTR gene in mouse models of ATTR, reducing serum TTR levels by up to 80%. This technology holds promise for treating not onlyATTR but also other incurable inherited diseases.
The Rice Brain Institute has awarded seed grants to four collaborative projects that unite Rice faculty with clinicians and scientists across the Texas Medical Center. The grants support innovative research in brain science, mental health, and neurological disease.
A University of California San Diego team is developing a first-of-its-kind stem cell-based gene therapy for Friedreich’s ataxia. The $7.4 million grant will support safety studies, manufacturing and clinical planning needed before applying to test the treatment in patients.
Researchers at Drexel University and UMass Chan Medical School have developed a gene therapy approach that silences and replaces disease-causing genes in mice with hereditary spastic paraplegia. The treatment prevents nerve breakdown and symptoms, offering a potential cure for the rare disease. Studies suggest that the therapy could be...
Waskyra, an ex vivo gene therapy, offers new hope for patients with Wiskott-Aldrich syndrome by reducing severe bleeding events and serious infections. The FDA approval confirms Fondazione Telethon's excellence in rare genetic disease research.
A study assessing the commercial roll-out of gene therapies for sickle cell disease and beta thalassemia reveals operational differences between two treatments. The median time to complete treatment was around 9-10 months, with most patients requiring only one cell collection procedure.
Preliminary results from trials of gene therapy exa-cel suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12. The therapy's potential to prevent irreversible complications makes it potentially more beneficial in children than adults.
Researchers at Rice University have developed a way to make serum markers more sensitive by editing them inside the bloodstream, enabling clearer observation of gene-expression changes in the brain. This approach could lead to more precise diagnostic capabilities using simple blood tests.
The review highlights the progress in gene-editing nucleases and delivery vectors for in vivo gene therapy, enabling targeted correction of genetic errors. Key examples include FDA-approved products targeting monogenic disorders and cancers, showcasing the therapeutic promise of combining genome editors with advanced delivery platforms.
Researchers have shown that disabling the NRF2 gene with CRISPR technology can restore drug sensitivity and slow tumor growth in lung cancer. The approach, which targets a master switch for resistance, has potential across multiple tumor types.
Researchers have discovered a new gene, ALOX15, that protects against sepsis-induced lung injury in mouse models. The findings suggest that mild lung thrombosis may actually reduce the severity of lung damage from sepsis, offering a potential novel therapeutic strategy.
A nonsurgical approach has been demonstrated to quiet a specific brain circuit in an animal model by delivering engineered gene therapy only to the targeted region. The method uses low-intensity focused ultrasound to open the blood-brain barrier, allowing precise control over brain activity without impacting off-target areas.
Researchers at Uppsala University have identified a new genetic technique that enables targeting of tumour cells with elevated levels of proteins driving medulloblastoma. The technique, which works like a Trojan horse, introduces an enzyme linked to SOX9 sequence, killing tumour cells.
A naturally occurring gene called Cyclin A2, normally silenced in humans, can make new functioning heart cells and aid in the heart's repair. The breakthrough discovery could lead to new techniques for repairing damaged hearts as an alternative to transplants or implanted cardiac devices.
Researchers at Weill Cornell Medicine have developed a powerful new gene-switch tool called Cyclone, which allows scientists to turn on or off target genes with precision. The tool uses a non-toxic molecule acyclovir to suppress gene activity, and has the potential to be adopted throughout biomedical research and gene therapies.