Research on treating disease by editing or delivering genes. Covers sickle-cell gene therapy, gene-editing nanoparticles, mRNA delivery, and blood-cell-inspired carriers.
In vivo CAR-T therapy reduces manufacturing time and cost, enabling more accessible cancer immunotherapy. The strategy also enables superior self-renewal capacity and anti-tumor persistence.
Researchers developed a gene-delivery system that converts reactive astrocytes into functional neurons, improving motor recovery in mice and rats. The system, TRANsCre-DIONE, selectively targets scar-forming cells and reprograms them into neurons, which generate nerve impulses and receive signals from other neurons.
The FDA approved FAYUVI, a gene therapy treatment for pediatric patients with Sanfilippo Syndrome Type A, following years of research at the Abigail Wexner Research Institute. The treatment, developed by Nationwide Children's Hospital, is the fourth gene therapy to gain FDA approval from the institution.
A new framework proposes that chronic overactivation of the DNA damage response may drive biological aging. Persistent DDR signaling can stabilize p53, increase p21 and p16INK4a, promoting cell-cycle arrest and senescence. This forms the basis of the
Scientists developed adenine base editing to correct a genetic mutation causing hereditary motor neuron disease. The therapy successfully alleviated neurodegeneration in a mouse model and patient-derived organoids, offering hope for precision gene therapies to address neurodegenerative diseases.
Researchers have developed a novel genome engineering method called prime assembly, which enables long DNA fragments to be stitched into precise target positions within living cells. This approach may allow for the development of universal gene therapies that can apply to many patients.
Researchers from Boston Children's Hospital have developed a new gene therapy approach that collects enough stem cells in a single hospital admission, improving turnaround time for genetically altering and infusing cells. The approach has shown long-term stability and safety, with patients not experiencing any adverse effects.
Researchers developed a bioinspired lipid nanoparticle that delivered gene-editing machinery to the liver, reducing low-density lipoprotein (LDL) cholesterol by over 20% and showing fewer signs of inflammation and toxicity. The nanoparticles also demonstrated positive effects on inflammation and healthy blood flow in cell experiments.
Researchers at Nagoya University developed a new delivery vehicle for circular RNA (cirRNA) using a novel lipid nanoparticle, FL0445-LNP, which improves the stability and efficacy of mRNA-based therapeutics. The technology has potential applications in cancer vaccines, genome editing, and protein supplements.
Researchers create engineered extracellular vesicles from red blood cell lipids, evading immune cells and targeting cancer cells. The technology offers flexibility in cargo loading and packaging, enabling delivery of genetic material, proteins, and whole viruses.
Researchers at UMass Chan Medical School have developed a microRNA-based gene therapy that suppresses mutant SOD1 production, delaying disease onset by 60 days and extending lifespan by 100 days in mice models of ALS. The therapy, delivered via adeno-associated virus (AAV) vector, preserves motor neurons and maintains neuromuscular con...
Research reveals that patients' inherited genetic variants can impact the benefits and toxicity of CAR-T cell therapy for blood cancers. Variants in genes such as STXBP2, ADAMTSL3, and PTPN22 were found to correlate with treatment-related toxicity or enhanced therapeutic activity.
Researchers developed a personalized gene therapy that dramatically reduced seizures and sparked developmental gains in two children with SCN2A-related DEE, allowing one to walk independently. The therapy targets the root genetic cause of the condition, producing measurable improvement.
Researchers at Mass General Brigham have been awarded $25.8 million in ARPA-H funding to develop a scalable gene editing platform for treating rare genetic vascular diseases. The VESSEL program aims to create durable, potentially one-time treatments for severe genetic vasculopathies.
The study demonstrated sustained improvements in disease features in mouse models with effects lasting throughout their lifespan. Genespire's approach has the potential to translate into human health as a single-administration treatment for patients with MMA.
Researchers have successfully produced genome-edited T cells that can target and destroy cancer cells using the Platinum TALEN genetic engineering technique. The technique was found to be efficient, with an average yield of 72 million 1G4-TCR cells for every 3 million T-cells.
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 at UCL and Great Ormond Street Hospital have successfully treated a deadly childhood liver disease using mice with a healthy version of the VPS33B gene. The treatment, which specifically targeted liver cells, showed no harm and improved liver function in mice.
Researchers developed a gene therapy that restored normal brain activity and improved behavior in mice with Fragile X syndrome by replacing the missing FMRP protein. The treatment administered during early development showed significant improvements in cognitive flexibility, social interactions, and probabilistic reversal learning.
Researchers at UT MD Anderson Cancer Center have achieved high response rates in patients with hard-to-treat acute myeloid leukemia (AML) using an all-oral combination therapy. The study also provides insights into the origins of cancer, revealing that tumors evolve rapidly through bursts of genetic changes.
A local research study found that only a minority of tested DNA switches were active in living liver tissue, with many linked to genes involved in metabolism and immune responses. The activity of these switches shifted in response to changes in the gut microbiome.
The University of Virginia has joined SPARK GLOBAL to leverage resources and expertise in accelerating the development of new medicines. This collaboration aims to reduce time from lab discoveries to clinic trials, delivering tangible healthcare solutions to patients.
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 research team at the University of Zurich has developed a new gene editing approach that correctly treats the genetic mutation causing hereditary epilepsy in mice. The therapy improves communication between nerve cells, reduces febrile seizures, and increases survival rates.
Researchers have developed a new gene editing technology called 'prime assembly' that allows efficient insertion of large DNA segments into the human genome. This innovation enables treatment of genetic diseases by replacing entire genes, promising substantial progress in treating conditions with multiple mutations.
A preclinical study using precision gene editing targets and corrects the root cause of Dravet syndrome, a rare form of childhood epilepsy. The approach demonstrates significant success in treating the disease, including improved survival rates and reduced seizures.
Toloo Taghian, a UMass Chan Medical School assistant professor, has received a five-year, $3.2 million grant to develop a gene therapy for UBA5 disorder, a rare genetic disease that affects protein balance and leads to neurological impacts. The goal is to monitor disease progression and determine the efficacy of future clinical trials.
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.
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.
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.
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.
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.
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.
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.