Articles tagged with Gene Editing
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The CRISPR Journal has published new research on gene editing regulations, introducing potential global implications for food animal production. A bioinformatic pipeline, dubbed CRISPRdisco, has been developed to help researchers identify and characterize CRISPR repeats and genes encoding Cas nucleases.
The inaugural issue of The CRISPR Journal features a range of articles on CRISPR biology, technology, and genome editing. Research highlights include progress in treating genetic diseases, such as hereditary blindness and Batten disease, using CRISPR-Cas9 gene editing.
The CRISPR Journal debuts with original research papers on plant editing, gene drives, and therapeutic applications. Researchers make progress in targeting specific mutations for inherited diseases like retinitis pigmentosa.
A new study found that person-to-person genetic differences can impact the efficacy of gene editing technologies like CRISPR-Cas9. The researchers analyzed 7,444 whole-genome sequences and discovered that about 50% of guide RNAs could be affected by variants at their target sites.
Experts discuss the potential risks and benefits of gene editing, including its applications in human health, agriculture, and the environment. The discussion highlights the need for harmonized policies across national borders to address concerns about misuse and unintended consequences.
Researchers successfully corrected a heart condition-causing mutation in human embryos, paving the way for potential treatments and prevention of inherited diseases. The technique uses CRISPR-Cas9 to target specific genetic mutations, offering hope for improving IVF outcomes and curing certain diseases.
A team of UC researchers is using gene editing to develop a new method to control disease-spreading mosquitoes, focusing on the Aedes aegypti mosquito. The Safe Genes project aims to introduce genetic elements that rapidly spread throughout a population, reducing the mosquito population and preventing disease transmission.
Researchers have identified an anti-CRISPR protein that can block the Cas9 component of CRISPR-Cas9 from interacting with DNA, reducing off-target cuts. This protein, AcrIIA4, was found to inhibit CRISPR-Cas9's ability to cut target DNA while still allowing on-target editing.
Researchers discovered anti-CRISPR proteins that decrease off-target side effects by up to four-fold, acting as a kill switch to disable CRISPR-Cas9 after its job is done. Delivering CRISPR and then the protein reduces off-target effects in human cells.
The CRISPR-Cpf1 gene editing system has been improved by incorporating a firefly gene, enabling the simultaneous targeting of multiple genes in human cells. This advance could be useful for treating diseases such as hepatitis B and muscular dystrophy.
Scientists used CRISPR/Cas9 gene editing to reverse Huntington's disease pathology and motor symptoms in a mouse model. The treatment delivered enzymes to brain cells, reducing toxic protein aggregates and improving motor abilities.
Scientists have demonstrated the effectiveness of gene editing in rhesus monkey embryos, paving the way for new therapies and treatments. The breakthrough opens up possibilities for human disease research, including neurological and reproductive conditions.
Actress Kiruna Stamell argues that gene editing raises significant ethical concerns, threatening social inequality and the adaptation of diverse communities. Dr. Christopher Gyngell counters that well-regulated gene editing can improve human health by reducing fatal genetic diseases.
Researchers at IBS prove the accuracy of a gene editing method that substitutes one nucleotide in the genome, finding it more accurate than CRISPR-Cas9. The technique caused fewer off-target changes, indicating its potential for widespread use.
Researchers envision AAV as a platform for delivering novel tools for genetic manipulation, including CRISPR-Cas9 and RNA interference. The combination of tailored delivery vectors and new editing techniques will enable unique approaches to therapeutic gene expression.
Researchers propose using CRISPR technology to domesticate sustainable and nutritious crops like wild legumes, quinoa, and amaranth. This approach aims to create 'biologically inspired organisms' by deleting existing genes instead of introducing new ones.
Researchers successfully produced the first transgenic mice with a single nucleotide difference in the dystrophin and tyrosinase genes, demonstrating a new gene editing technique that can substitute one nucleotide into another without DNA deletion. This breakthrough could potentially lead to the correction of genetic defects in humans.
Genome editing enables precise genetic changes in livestock, boosting productivity and reducing greenhouse gas emissions. Researchers aim to apply this technology to improve animal welfare and potentially produce single-gender offspring.
The ethics of gene editing is being explored in a session at the AAAS annual meeting, examining concerns beyond safety, such as modifying the human germline, parental relationships, and respect for persons with disability. Experts will discuss new technologies and their social and ethical implications.
A 258-page report outlines principles and guidelines for human genome editing, emphasizing caution on germline editing and enhancement. The report also explores clinical applications, risks, and benefits of the technology.
Scientists have developed a method to observe gene editing in real-time, enabling the investigation of CRISPR-Cas9's biological processes and potential applications for treating genetic diseases such as sickle cell anemia and cystic fibrosis.
A team of researchers discovered that a specific mutation in the telomere protein TPP1 causes an incurable premature aging disease called dyskeratosis congenita. The mutation compromises telomerase function, leading to stem cell division slowdowns and tissue breakdown. This breakthrough provides a potential drug target for the disease.
A new gene-editing system successfully cured a genetic blood disorder in living mice, offering a minimally invasive treatment for beta thalassemia and sickle cell disease. The technology significantly decreases unwanted gene mutations and uses FDA-approved nanoparticles to deliver PNA molecules.
Researchers developed a novel gene editing strategy to correct thalassemia mutations in mice, alleviating symptoms and normalizing hemoglobin levels. The technique, which uses nanoparticles and synthetic DNA, has the potential to treat people with inherited blood disorders like sickle cell anemia.
Researchers at St. Jude Children's Research Hospital have found a way to use CRISPR gene editing to help fix sickle cell disease and beta-thalassemia in blood cells isolated from patients. The study provides proof-of-principle for a new approach to treat common blood disorders by genome editing.
Researchers outline recent progress in clinical applications of retinal gene replacement therapy and preclinical advances in gene-specific therapy for photoreceptor diseases. Gene therapy strategies are being developed to treat a broader range of disorders affecting vision, providing new hope for individuals with eye diseases.
Promising results from clinical trials of globin gene transfer have eliminated the need for blood transfusions in some individuals with beta-thalassemias. Gene editing technologies hold promise to correct beta-globin deficiencies and reactivate fetal hemoglobin production, potentially leading to a cure for severe globin disorders.
The Hastings Center has launched a three-year project to examine the fundamental questions of using gene editing in humans, including its impact on human flourishing and core values. The project aims to develop new scholarship, equips journalists, and prepares teachers to engage with these issues.
Researchers suggest using CRISPR-Cas9 technology to introduce a male sex determining gene into female mosquito embryos, potentially reducing disease transmission. The goal is to create sterile or fertile males that outcompete females in the wild.
Researchers have developed a CRISPR-Cas9 gene editing system to treat Duchenne muscular dystrophy (DMD), a debilitating genetic disease. By deleting exon 23 and restoring dystrophin protein levels, the therapy improved muscle function in mice with DMD, including cardiac and pulmonary health.
Scientists have created RNA-based drugs that can temporarily activate and inactivate the CRISPR/Cas9 gene editing system, providing more precise control over gene expression. This breakthrough enables researchers to correct and inactivate genes with increased efficiency and potential selectivity.