Articles tagged with Gene Editing
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 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 have developed an ultracompact gene activation tool called TIGRa, which can target multiple genes simultaneously and is smaller than CRISPR-based tools. TIGRa was tested in a mouse model and showed promising results in treating retinal degenerative conditions, including glaucoma.
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 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...
Emerging non-endocytic delivery strategies enable direct cytosolic delivery of proteins, nucleic acids, and gene-editing tools, providing new opportunities for biomedical therapies. The review discusses their application prospects in gene therapy, macromolecular drug delivery, and cellular engineering.
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.
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.
The 2026 Warren Alpert Foundation Prize was awarded to five scientists who developed curative genetic therapies for two major blood disorders. These treatments, CASGEVY and LYFGENIA, have been approved by the FDA for use in the US and have shown significant advancements in the field of gene therapy.
Researchers developed a novel fluorescent-based method to analyze upstream open reading frames (uORFs) in plant genomes, enabling rapid analysis of gene regulation. The technique simplifies current methods by using intact leaf tissue and fluorescent proteins, reducing sample preparation and consumable materials.
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.
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 have discovered that plant viruses can deliver CRISPR RNA guides to plants, enabling targeted genome edits. The study showcases the potential of potyvirus-based systems for expanding crop genome editing.
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.
The SMArT platform achieves near-pure selection of correctly edited blood stem cells while reducing dangerous genomic alterations. The innovative strategy enables enrichment of cells with targeted integration to 100% purity.
Researchers successfully created a novel, stably inheritable grass carp germplasm without intermuscular bones using the runx2b gene. The absence of IBs did not affect the fish's quality or nutritional profile, but improved gel strength and resilience were observed.
Philip Tai, PhD, received a $1.6 million grant to investigate AAV vector mechanisms using high-resolution DNA sequencing technology. His findings could lead to new vector designs that improve gene therapy treatments' safety. The goal is to remove mutations that cause cancer-causing integration into host cells.
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.
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.
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.
Researchers summarize universal mechanisms of regulatory T cells in solid organ transplantation, enabling durable immune tolerance and reducing rejection. Gene-editing technologies create hypoimmunogenic Tregs for standardized, scalable availability.
A new CRISPR protein, Cas12a2, has shown potential for killing sick cells while leaving healthy ones untouched. Researchers have tested its effectiveness in destroying cancer cells and virus-infected cells with promising results.
Researchers at Gladstone Institutes identified hundreds of human genes influencing HIV infection and two potent antiviral proteins, PI16 and PPID. These proteins block HIV's entry into T cells or limit its ability to replicate within the cell.
Researchers have made a breakthrough in developing a novel platform that harnesses the immune system's ability to produce therapeutic proteins. By editing hematopoietic stem cells with CRISPR gene-editing tools, they were able to create a long-term, boostable source of antibodies capable of protecting against deadly influenza infection...
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.
A gene-edited treatment has shown remarkable success against severe sickle cell disease, with 27 out of 28 patients achieving a functional cure and no painful crises. The therapy uses CRISPR/Cas12a technology to modify stem cells and increase levels of fetal hemoglobin.
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.
Researchers have discovered an RNA-guided CRISPR system that can activate genes without cutting DNA, opening up new possibilities for gene regulation and therapeutic strategies. The system uses a strand of RNA as a guide to recruit the cell's transcription machinery, allowing for precise control over gene expression.
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.
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.
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.
A new rice gene, GSN7, has been discovered that can simultaneously increase grain yield and improve quality. The study found that precise manipulation of this gene can overcome the long-standing trade-off between yield and quality in rice breeding.
UCLA researchers have developed a novel gene-editing approach using lipid nanoparticles to deliver a full-length CFTR gene into human airway cells. The study shows promise for treating cystic fibrosis by correcting the underlying genetic mutation, which could lead to more effective and long-term therapies.
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 developed a gene-editing therapy that directly corrects genetic mutations responsible for autosomal dominant polycystic kidney disease, slowing cyst growth and improving health outcomes. The therapy uses CRISPR-based base editing to precisely correct single-letter DNA mutations in the PKD1 gene.
Researchers at the University of British Columbia have developed a topical CRISPR-based therapy that can correct faulty genes in human skin, potentially treating genetic skin conditions like ARCI and eczema. The treatment, using lipid nanoparticle technology, restores up to 30% of normal skin function.
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.
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.
Researchers at St. Jude Children's Research Hospital created CHANGE-seq-BE to evaluate the activity and specificity of base editors, a genome editing technology, ensuring safety and accuracy. The technique outperformed conventional approaches and has already been used in clinical work.
A new treatment using genome-edited immune cells has shown promising results in helping children and adults fight a rare form of blood cancer called T-cell acute lymphoblastic leukaemia (T-ALL). The gene therapy, known as BE-CAR7, uses base-editing to modify T-cells and destroy cancerous cells.
Researchers have made significant advances in genetically modified plants that produce alpha-amylase inhibitor proteins, making them indigestible to pests like bedbugs, beetles, weevils, and woodworms. Gene editing techniques, such as CRISPR, offer a promising solution to combat insect pests without compromising human consumption.
Plant biologists developed a method to grow transgenic plants in weeks instead of months by hijacking a plant's natural regeneration abilities. The technique uses bacteria carrying genetic instructions for wound healing and regeneration to trigger plant growth.
Researchers at the University of Texas at Austin have developed a novel gene-editing method that can correct multiple disease-causing mutations simultaneously. This approach uses bacterial retrons to protect the microbes from viral infection and has shown promising results in correcting scoliosis-causing mutations in zebrafish embryos.
Researchers create enhanced T cells with improved survival in cancer models by simultaneously modifying multiple genes using CRISPRoff and CRISPRon. The approach overcomes toxicity issues associated with traditional gene editing methods, enabling high cell survival rates and potential for treating various diseases.
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...
A pioneering case of pig-to-human liver xenotransplantation has been successfully demonstrated, with the genetically engineered porcine liver functioning for an extended period in a human recipient. The patient survived for 171 days despite complications such as xenotransplantation-associated thrombotic microangiopathy.
Researchers at CNIO have created a 'human repairome', a catalogue of 20,000 DNA 'scars' that reveal how genes affect DNA repair. This information can help determine the best treatment for each cancer type and overcome resistance to therapy.
A UCLA research team led by Dr. Donald Kohn has developed a one-time stem cell gene therapy treatment for alpha thalassemia major that could be curative. The therapy involves adding the missing alpha-globin gene to patient cells using a viral vector, enabling them to produce functional hemoglobin.
Mass General Brigham has identified key areas of innovation in medicine, including gene editing, immune system modulation, AI-powered care, and organ transplantation. The 'Big Ideas in Medicine' aim to transform diagnosis, treatment, and prevention of disease with next-generation technologies.
Researchers found that mutations in the CFAP410 gene change its interaction with another protein, making motor neuron cells more vulnerable to DNA damage and cell death. This discovery provides new insights into the mechanisms underlying Motor Neurone Disease and highlights potential targets for new therapies.
Researchers at Northwestern University have developed a new CRISPR delivery system that triples efficiency using DNA-wrapped nanoparticles, improving safety and effectiveness. The new system, called LNP-SNAs, targets specific cells and tissues, reducing toxicity and boosting gene-editing efficiency by threefold.
Researchers at EPFL developed BindCraft, an open-source AI platform that uses AlphaFold2 to generate novel binders with desired functional properties. The platform reduces the need for high-throughput screening and makes protein design more democratized.
A study published in the Journal of Hepatology reveals that only 15-20% of neonatal liver cells are responsible for generating over 90% of the adult liver mass. This finding has major implications for pediatric gene therapy, allowing scientists to achieve more effective and durable correction of inherited liver diseases.
Researchers have successfully treated damage caused by heart attacks in non-human primates using gene therapy, restoring both strength and rhythm of the damaged hearts. The treatment improved heart function in pathological conditions with no adverse effects observed.