Articles tagged with Gene Editing
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.
Researchers have used CRISPR gene editing to study the regulation of the Unusual Floral Organs (UFO) gene in plants, uncovering the importance of conserved non-coding DNA sequences in controlling flower formation.
Researchers use CRISPR-Cas9 to remove duplicate chromosomes in trisomy 21 cells, restoring gene expression and cellular phenotypes. The technique shows promise as a potential medical intervention for people with Down syndrome.
Researchers from Flinders University applied gene editing to explore the role of enzyme ACE2 in healthy placental development. They found that ACE2 plays a key role in helping cells grow properly and that a genetic variation is linked to major pregnancy complications.
Researchers successfully developed CoQ10-producing rice through targeted gene editing, offering a cost-effective approach to nutritional fortification. The discovery provides great potential benefit for human health, particularly heart protection, and expands the food sources of CoQ10.
Researchers at UCSF used CRISPR gene editing technology to transform ordinary white fat cells into 'beige' fat cells that voraciously consume calories to make heat. Implanted near tumors, these cells outcompeted cancer cells for nutrients, beating back five types of cancer in lab experiments.
Scientists create complex human cell lines with random genome structural changes to study their impact on cell survival and gene expression. They found that essential genes must remain intact for significant structural changes to be tolerated.
UCSF researchers found that when brain cells of female mice express only a maternal X chromosome, their memory and cognitive skills deteriorate faster. This study could explain the variation in brain aging between the sexes and individual women.
Researchers have developed a method for delivering therapeutics to targeted locations in the human body using universal milk exosomes. This technology could be used to treat common and rare diseases alike, offering a potential boon to rare disease communities.
A recent study from the University of Illinois shows that gene-edited bacteria can supply equivalent of 35 pounds of nitrogen from air during early corn growth, increasing vegetative growth, nitrogen accumulation and yield by an average of 2 bushels per acre.
Researchers developed a gene-editing treatment that reduces prion protein levels in the brain by up to 60% and extends mouse lifespan by about 50%. The base-editing approach could lead to a one-time treatment for prion disease patients.
Lehigh University bioengineering researcher Tomas Gonzalez-Fernandez is exploring how combining CRISPR with biomaterials can improve gene editing's safety and efficacy for therapeutic use. His NSF CAREER award-funded research aims to develop more targeted and controlled therapies for genetic diseases.
Researchers have developed a new genetic engineering tool, mvGPT, that can precisely edit genes, activate gene expression, and repress genes all at the same time. The technology has shown promise in treating genetic diseases such as Wilson's disease and type I diabetes by targeting multiple genetic conditions simultaneously.
Researchers developed a gene therapy approach to treat chronic hypereosinophilia by delivering an anti-human eosinophil antibody via AAV-based gene therapy. The therapy successfully suppressed blood eosinophil levels in mice, showing promise as a potential treatment for the condition.
A new gene editing tool called SPLICER has been applied to reduce the formation of amyloid-beta plaque precursors in a mouse model of Alzheimer's disease. The application shows improved efficiency over current standard gene editing technology and potential for application in other diseases.
Researchers compared urine NAA levels in patients with mild and typical Canavan disease, finding lower levels in those with the milder form. This discovery has potential for a rapid and cost-effective way to screen for CD incidence and severity.
A USC Stem Cell study has identified key gene regulators that enable some deafened animals, including fish and lizards, to naturally regenerate their hearing. The researchers found a class of DNA control elements known as 'enhancers' that amplify the production of a protein called ATOH1, which induces sensory cells in the inner ear.
Branden Baptiste, 20, becomes the world's first patient to receive base editing gene therapy for sickle cell disease. The treatment has left him feeling 'more than fine' with no symptoms of the disease. After undergoing chemotherapy and a series of tests, Branden was infused with genetically treated cells in December 2023, allowing his...
A new study found that gene therapy delandistrogene moxeparvovec significantly extended the median survival of Duchenne muscular dystrophy (DMD) rats to >25 months. Additionally, the treatment elicited statistically significant improvements in cardiac parameters and mobility.
Researchers have discovered a major setback in the use of AZD7648 to promote precise gene editing, which causes massive genetic changes and genome instability. Despite this, scientists remain optimistic about advancing CRISPR-Cas technology to treat diseases.
Engineers at USC Viterbi School of Engineering have developed a new CRISPR toolkit that allows for precise, remote-controlled genome editing using focused ultrasound. This breakthrough enables the treatment of various genetic disorders and diseases by activating or silencing specific genes with precision.
Researchers at UCSF are enrolling patients in a clinical trial to correct the genetic mutation causing sickle cell disease using non-viral CRISPR-Cas9 gene editing. The therapy aims to eliminate the need for a bone marrow transplant and create a new blood system free of the disease.
A new gene drive technology, known as e-Drive, has been developed to reverse insecticide resistance in pests by replacing mutant genes with native ones. The system is designed to spread and then disappear, leaving only a population of insects susceptible to pesticides.
Researchers developed a novel lentivirus-based gene therapy strategy in CD34+ hematopoietic progenitor cells, which showed therapeutic levels of expression of the anti-sickling beta globin protein. Cyclosporin improved transduction efficiency and preserved cell viability.
Researchers have developed a new CRISPR-Cas method to decipher the function of genetic variants that contribute to cancer. The approach creates tens of thousands of cells with different gene variants, allowing scientists to identify which variants make cancer cells resistant to standard drugs.
A new study uses CRISPR-Cas13 to identify nearly 800 noncoding RNAs that are functional and essential for cell function, including in cancer and human development. The researchers found that these RNA molecules modulate key pathways for cell proliferation and can serve as potential biomarkers and therapeutic targets for cancer treatment.
The CRISPR tool was successfully used to correct a genetic defect in cells affected by chronic granulomatous disease. However, the repair process also introduced new genetic defects, highlighting the need for caution when using CRISPR technology in clinical settings.
Using state-of-the-art technology, researchers identified specific steps needed for CRISPR to become active and perform its gene editing function. Understanding these mechanisms could lead to improved designs for CRISPR-based gene editing.
Researchers used ex vivo lentiviral gene therapy to treat MPS IVA in mice, achieving partial correction of bone pathology and complete correction of heart pathology. The study suggests potential for novel therapies to treat patients with MPS IVA.
A CRISPR-Cas9 based gene editing therapy has been shown to reduce angioedema attacks and sustainably lower kallikrein levels in patients with hereditary angioedema, offering a potential treatment option. The therapy was tested on 27 patients across two dosages compared to placebo.
A USC Stem Cell mouse study identifies a small subset of blood stem cells as the primary driver of immune aging. The researchers found that this subset overproduces innate immune cells, leading to an age-associated imbalance and increased disease risk. By targeting this subset, the study suggests a potential therapy to delay immune agi...
Dr. Fyodor Urnav proposes a set of initiatives to address the crisis, including pooling patients by syndrome and permitting multiple gene editors in a single Investigational New Drug application. This approach aims to accelerate the development of CRISPR therapies for rare genetic diseases.
Researchers at ChristianaCare Gene Editing Institute use CRISPR tools to safely disable gene mutation linked to treatment-resistant melanoma. The approach targets melanoma tumor cells while leaving healthy cells alone, restoring sensitivity to anticancer drugs.