Gene Editing
Articles tagged with Gene Editing
Hereditary epilepsy successfully treated in mouse model
UMass Chan scientists develop gene editing technology capable of rewriting entire chapters of the genome
Toloo Taghian awarded $3.2M to develop gene therapy for a rare genetic disease
From immunosuppression to active tolerance: the evolving paradigm of regulatory T cell therapy in organ transplantation
New kind of CRISPR could treat viral infection and cancer by shredding sick cells’ DNA
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.
Scientists map how HIV hijacks human cells—and how cells can fight back
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.
Programming the immune system to manufacture its own therapeutic proteins
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...
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.
Gene editing therapy shows success against severe sickle cell disease
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.
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.
RNA-guided CRISPR system activates gene expression
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.
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.
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.
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.
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.
New rice gene breaks the yield–quality trade-off in rice
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.
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.
Nanoparticle-based gene editing could expand treatment options for cystic fibrosis
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 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.
Mayo Clinic uses gene editing to reverse inherited kidney disease in preclinical study
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.
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.
Scientists develop first gene-editing treatment for skin conditions
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.
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.
CHANGE-seq-BE finds off-target changes in the genome from base editors
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.
‘Ready-made’ T-cell gene therapy tackles ‘incurable’ T-cell leukemia
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.
Gene editing produces plants that are indigestible to pests
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.
Growing transgenic plants in weeks instead of months by hijacking a plant’s natural regeneration abilities
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.
New gene-editing tech holds promise for treating complex genetic diseases
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.
Epigenetic reprogramming safely modifies multiple genes in T Cells simultaneously for CAR-T therapies
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.
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...
World’s first pig-to-human liver xenotransplant in a living recipient reported in the Journal of Hepatology
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.
CNIO researchers create the “human repairome”, a catalogue of DNA “scars” that will help define personalized cancer treatments
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.
UCLA scientists advance gene therapy for deadly blood disorder alpha thalassemia major
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.
The ‘Big Ideas in Medicine’: Mass General Brigham leaders identify top opportunities to impact health care
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.
New study uncovers how DNA damage can lead to Motor Neurone Disease
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.
CRISPR’s efficiency triples with DNA-wrapped nanoparticles
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.
An open-source AI platform to democratize protein design
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.
Clonogenic hepatocytes drive postnatal liver growth and unlock new avenues for pediatric gene therapy
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.
Gene therapy restores functionality in non-human primates after heart attacks
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.
U. Iowa research identifies promising new target for treating rare, aggressive childhood cancer
Researchers at U. Iowa identify a specific metabolic pathway that fuels tumor growth in MPNST, providing a promising new target for treatment. By blocking this pathway, tumors grow more slowly and are more vulnerable to chemotherapy.
Laying the foundation for gene editing for inherited progressive deafness in adults, DFNA41
Researchers developed a one-time gene editing treatment that restored hearing and balance in adult mice with DFNA41, a genetic form of progressive deafness. The therapy successfully disabled the harmful mutation while preserving the normal gene, leading to long-term hearing and balance restoration.
Scientists propose AI-driven biotech model for future crop breeding
A team of scientists proposes an integrated framework combining biotechnology and AI to revolutionize crop breeding, exploring multi-omics, genome editing, and high-throughput phenotyping. The authors present a forward-looking framework for AI-assisted crop germplasm design, offering a roadmap for sustainable agriculture.
Stealth genetic switch in mosquitoes halts malaria spread
Researchers developed a CRISPR-based gene-editing system that changes a single molecule within mosquitoes, halting malaria-parasite transmission. The new system is designed to genetically spread the malaria resistance trait until entire populations of the insects no longer transfer the disease-causing parasites.
Evolution's gamble: Human-specific genes that shaped human intelligence may fuel cancer
A recent study identified 37 human-specific genes linked to brain development and cognitive abilities, which are hijacked by cancer to drive tumor growth. The researchers discovered nearly half of these genes become aberrantly activated in cancerous tissues, promoting tumor cell proliferation.
$2.6 million NIH grant backs search for genetic cure in deadly heart disease
Scientists are working on a genetic cure for the world's most common inherited heart disease, hypertrophic cardiomyopathy (HCM), with a new gene editing tool. The team aims to correct mutations that cause the disease, which affects 14 million people worldwide.
Synthetic DNA nanoparticles for gene therapy
Researchers at Case Western Reserve University have received an NSF CAREER Award to develop synthetic DNA nanoparticles with potential applications in gene therapy. The program aims to study how these particles behave inside cells and potentially design therapeutics for genetic diseases.
Scientists use gene editing to correct harmful mitochondrial mutations in human cells
Researchers have successfully edited harmful mitochondrial DNA mutations in liver and skin cells using a genetic tool called a base editor. The study, published in PLOS Biology, offers promising results for treating mitochondrial diseases and aging-related conditions.
National UCD Foundation to build network, create roadmap for future research in urea cycle disorders
The National Urea Cycle Disorders Foundation is establishing a multistakeholder Partner Network to guide health care decisions and build a sustainable infrastructure for UCD research. The project aims to empower the broad UCD community to work together effectively, identify research needs, and create a roadmap for future studies.
KAIST develops technology for selective RNA modification in living cells and animals
Researchers at KAIST have developed a groundbreaking technology capable of selectively acetylating specific RNA molecules within the human body using the CRISPR-Cas13 system. This breakthrough enables precise, programmable control of RNA function and is expected to open new avenues in RNA-based therapeutic development.
Stress genes clear dead cells, offering disease insights
Researchers have discovered a novel cell-clearance pathway linked to diseases such as Chediak-Higashi Syndrome, which affects immune system function. The study used CRISPR/Cas9 gene-editing technology and live imaging to characterize this pathway and identify key genes involved.
A better understanding of how gene editing tools work
Researchers discovered that a ligase called Lig3 inhibits base editing, while the mismatch repair pathway helps cytosine base editing. The study sheds light on the complex mechanisms behind base editing and its potential applications in treating genetic diseases.
New insights into long-term dysfunction of edited blood stem cells and how to overcome it
Scientists at San Raffaele Telethon Institute for Gene Therapy discovered that CRISPR-Cas9 gene editing can cause inflammation and senescence-like responses in blood stem cells. This reduces the cells' ability to regenerate blood cells after transplantation, limiting the long-term success of gene therapy.
Gene editing disrupts Huntington’s mutation in mice
Researchers at the Broad Institute developed a gene editing approach that interrupts and stabilizes trinucleotide repeat expansions, which cause Huntington's disease and Friedreich's ataxia. The method, using base editing, prevents the repeats from growing in length, halting or slowing down disease progression.
Researchers develop gene therapy that can target airway and lungs via nasal spray
Researchers developed a gene therapy that can target the airway and lungs using a nasal spray, outperforming previous versions in preclinical models. The innovative tool, AAV.CPP.16, showed promise for treating respiratory diseases like pulmonary fibrosis and viral infections.
World's first patient treated with personalized CRISPR gene editing therapy at Children’s Hospital of Philadelphia
A child diagnosed with a rare genetic disorder has been successfully treated with a customized CRISPR gene editing therapy, showcasing the power of tailored gene editing to treat patients. The infant is now growing well and thriving after receiving three doses of the therapy with no serious side effects.
New gene editor enables greater precision
The evoCAST system enables precise insertion of entire genes into the human genome, overcoming a major challenge in gene therapy. This breakthrough could lead to more reliable treatments for diseases like cystic fibrosis and hemophilia.
Mass photometry is the key to exciting advances in gene therapy manufacturing
Researchers from The University of Osaka developed a new technique using mass photometry to detect and quantify components of rAAV particles. This method can distinguish between full and empty particles, streamlining gene therapy manufacturing and improving clinical effectiveness.