Articles tagged with Gene Editing
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.
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 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.
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.
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 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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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 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.
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.
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.
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.
Researchers from Mass General Brigham presented key findings from multiple innovative studies on gene and cell therapy, focusing on rare diseases, brain cancer, and neurodegenerative disorders. The studies explored strategies to improve care delivery and accelerate translation from lab to clinic, with potential breakthroughs in treatin...
Researchers have successfully tested a CRISPR/Cas9 gene-editing technique to enhance the immune system's fight against advanced gastrointestinal (GI) cancers. The treatment showed encouraging signs of safety and potential effectiveness in patients with stage IV colorectal cancer, halting tumor growth and even achieving complete responses.
Researchers have developed a new method using CRISPR to identify microorganisms in natural environments, providing a clearer picture of the communities present. This breakthrough could lead to a better understanding of how microbes support ecosystem health and promote sustainable agriculture.
Researchers have developed a new method to genetically improve poplar trees without introducing foreign DNA into its genome. This breakthrough could accelerate the deployment of genetically enhanced trees with benefits for both the environment and the bio-based economy.
A new machine learning model accurately predicts the fitness of AAV capsids based on their amino acid sequence, enabling more efficient and cost-effective gene therapies. The model's robustness and generalizability have been demonstrated through tests on independent datasets, offering a promising tool for capsid engineering.
STITCHR uses an RNA system to replace entire genes, overcoming CRISPR limitations in targeting every mutation. The tool offers a one-and-done approach for gene therapy, addressing cystic fibrosis and other diseases with thousands of mutations.
Researchers have developed a nonviral gene delivery system to introduce transgenes into cynomolgus monkeys, expanding the use of genetic models for human disease research. The piggyBac transposon system allows for precise selection of modified embryos and flexible expression control.
A team from the University of Illinois found that traditional breeding methods are unlikely to improve soybean light-harvesting efficiency. Gene editing is likely needed to unlock soybean potential. The researchers gathered detailed measurements throughout an entire growing season to understand photoprotection relaxation in soybeans.
A new study found that recombinant adeno-associated virus (rAAV) capsids contain single-stranded DNA impurities derived from plasmid and host cell DNA. The researchers suggest that the adverse effects of these impurities may differ from those of double-stranded DNA, highlighting the need for further evaluation.
Genetic changes triggered by environmental factors like pollution, diet, and stress can increase cancer risk. Nearly everyone is exposed to cancer risk factors daily, highlighting the need for public awareness and policy action to reduce exposure.
Researchers used CRISPR to cut a single gene from cancer cells of head and neck tumors, resulting in the elimination of 50% of the tumors after 84 days. This groundbreaking study demonstrates that some genes are essential for cancer cell survival, making them excellent targets for CRISPR therapy.
A study published in Human Gene Therapy found that over half of individuals with Niemann-Pick disease type C1 lacked neutralizing antibodies against AAV2 and AAV9. This absence of antibodies may impact the effectiveness of gene therapy treatments for this rare disorder.
Researchers developed gene-edited lettuce with significantly higher levels of β-carotene, zeaxanthin, and ascorbic acid without compromising plant growth. This breakthrough highlights the potential of gene editing to combat micronutrient deficiencies and improve dietary quality.
The article discusses the need for bioanalytical assays to measure immune responses to oligonucleotide therapeutic drugs, especially when they include carriers or conjugates. Highly specific antibodies may enhance the development and production of ONTs, expanding studies on their safety and efficacy.
Sylvester's Firefighter Cancer Initiative reduces cancer risk among firefighters and community members by addressing electric vehicle fires. A new gene-editing study aims to treat mitochondrial diseases, while a program connects patients with pathologists to improve understanding of complex results.