Gene Therapy
Articles tagged with Gene Therapy
Precision DNA editing targets root cause of severe childhood epilepsy in preclinical study
Hereditary epilepsy successfully treated in mouse model
Toloo Taghian awarded $3.2M to develop gene therapy for a rare genetic disease
New technology enables ‘rewriting a chapter’ of the genome
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.
$13 million CIRM award to fund research on the role of RNA pollution in neurodegenerative diseases
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.
Building a better delivery system for gene editing machines by re-engineering the cellular factory
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.
Cystic fibrosis: research advances
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.
Hearing restoration from gene therapy for inherited deafness lasts years, new trial results show
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.
Stuart Orkin receives Breakthrough Prize in Life Sciences
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.
In mice, gene editing repairs a mutation that causes rare liver disorder
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.
New bite-sized CRISPR molecule may open doors for therapeutic genome editing
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.
Scientists reverse severe epilepsy in lab mice in promising step toward a cure
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.
Genetically modified marmosets as a model for human deafness
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.
Nanoparticles genetically modify several human cell types
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.
FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder
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.
USC receives funding for AI tool to advance treatment for rare pediatric diseases
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 could help the heart repair itself
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.
Researchers develop new way to safely insert gene-sized DNA into the genome
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.
Gene therapy for Duchenne muscular dystrophy: Genethon confirms two-year efficacy in patients treated with its drug candidate GNT0004 at therapeutic dose in the first phase of its clinical trial
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.
Atamyo Therapeutics presents promising results in the first patients treated with its ATA-200 gene therapy in the clinical trial targeting LGMD-R5 limb-girdle muscular dystrophy
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.
First gene regulation clinical trials for epilepsy show promising results
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.
Platform to map living brain noninvasively takes next big step
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 sharpen gene-editing tools to target cystic fibrosis
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.
Scientists deliver new molecule for getting DNA into cells
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.
Klaudia Kuranda joins Genethon and the Institute of Myology to create a center of expertise dedicated to immunology
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.
New Lund model aims to shorten the path to life-saving cell and gene therapies
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.
Noninvasive paths to complex brain science: Rice bioengineer named Sloan Research Fellow
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: a comprehensive review of pathogenesis, diagnosis, and management
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.
Cell and gene therapy across 35 years
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 to deliver keynote address at ISSCR 2026 Annual Meeting in Montréal
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 appointed chair of UC Davis Department of Biochemistry and Molecular Medicine
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.
From cytoplasm to nucleus: A new workflow to improve gene therapy odds
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.
AAVLINK: Potent DNA-recombination method for large cargo delivery in gene therapy
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.
RNA therapy may be a solution for infant hydrocephalus
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 addresses access and affordability in cell and gene therapies; convenes summit on 20 March 2026
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.
Could gene therapy treat a deadly heart condition that targets young athletes?
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.
Caring Cross and Boston Children’s Hospital collaborate to expand access to gene therapy for sickle cell disease and beta thalassemia
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.
Rice bioengineers explore new gene delivery systems with Keck Foundation support
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.
Genethon signs licensing agreement with AskBio for the development of an investigational gene therapy for Pompe disease
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.
New study explores therapeutic potential of CRISPRCas3 genome-editing system
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.
Rice Brain Institute awards first seed grants to jump-start collaborative brain health research
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.
California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’s ataxia
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.
Breakthrough on gene therapy for hereditary spastic paraplegia
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...
Fondazione Telethon announces FDA approval of Waskyra™ (etuvetidigene autotemcel), a gene therapy for the treatment of Wiskott-Aldrich syndrome
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.
Early results suggest exa-cel gene therapy works well in children
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.
Study offers real-world data on commercial implementation of gene therapies for sickle cell disease and beta thalassemia
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.
A molecular ‘reset button’ for reading the brain through a blood test
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.
Gene therapy: A new frontier against genetic mutations, degenerative diseases, and cancers
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.
Study: New potential for gene-based treatment for sepsis and lung injury
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.
CRISPR breakthrough reverses chemotherapy resistance in lung cancer
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.
Nonsurgical treatment shows promise for targeted seizure control
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.
New technology may enable precision treatment of pediatric brain tumors
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 specific human gene can help the heart repair itself from heart attack or heart failure
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 unveil a powerful new gene-switch tool
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.
Guidelines for treating hereditary hearing loss with gene therapy from international experts
International experts have established guidelines for gene therapy in hereditary hearing loss, emphasizing patient-centered care and respect for diversity. The guidelines outline frameworks for clinical trials, including patient selection criteria, surgical procedures, and safety evaluations.
Landmark gene therapy study shows safety for children
A landmark study published in the New England Journal of Medicine reports the long-term safety and efficacy of gene therapy for children with ADA-SCID, a rare immune disorder. The treatment resulted in a 100% survival rate and over 95% cure rate, with patients able to respond to routine childhood vaccinations.
Gene therapy delivers lasting immune protection in children with rare disorder
A new gene therapy has restored and maintained immune system function in 59 of 62 children born with ADA-SCID, a rare genetic immune disorder. The treatment involves delivering a healthy copy of the ADA gene to patients' blood stem cells, allowing them to produce healthy immune cells capable of fighting infections.
MIT researchers develop a new system can dial expression of synthetic genes up or down
Researchers at MIT have developed a new system that allows for precise control over the expression of synthetic genes in cells. The DIAL system uses a promoter editing mechanism to establish desired protein levels, which can be edited after delivery. This technology has the potential to improve gene therapy and cell reprogramming appli...
Scientists fix genetic defect in mice tied to brain disorders that include autism and epilepsy
Researchers developed a new gene therapy that reversed symptoms related to SYNGAP1-related disorders in mice, including intellectual disability, epilepsy, and risk-taking behaviors. The therapy successfully delivered a working copy of the SYNGAP1 gene into brain cells using an adeno-associated virus, offering hope for treatment in humans.