Articles tagged with Gene Editing
Researchers at the University of Iowa have made significant breakthroughs in delivering gene-editing proteins to airway cells without causing harm. The new peptide-based platform shows promise in treating diseases like cystic fibrosis, COPD, and asthma by repairing or modifying disease-causing mutations.
Scientists have quantified the rate of Cas9-caused off-target mutagenesis in mice through whole-genome sequencing, revealing that guide RNA design can significantly reduce unwanted mutations. The study highlights the need for improved precision in gene editing, particularly in therapeutic applications.
The Carnegie Mellon and Yale research team will scale up production of PNAs, improve DNA binding properties, and develop new nanoparticle formulations for enhanced in vivo editing. The goal is to move the technique closer to clinical therapeutic applications.
Researchers have developed a new CRISPR-Cas9 variant that reduces unintended changes in DNA, increasing precision in gene therapy. The SaCas9-HF variant shows high on-target efficiency and nearly undetectable off-target activity, offering a promising alternative for precise genome editing.
Scientists Zach Lippman and Yuval Eshed review past agricultural revolutions, highlighting key genetic mutations and modifications. They propose using CRISPR gene editing to introduce new variations in core hormonal systems, potentially boosting crop productivity and adaptability.
The book explores the social and ethical implications of gene editing on human germline cells, including its impact on relationships between parents and children, health, normalcy, and well-being. Leading thinkers weigh in on the potential risks and benefits of this revolutionary technology.
Scientists successfully edited the genes of albino lizards using CRISPR-Cas9, allowing them to study gene regulation and vision development. The technique, which can be applied to other animals, demonstrates a new approach for manipulating reptile genomes.
The CRISPR Journal has published new articles on iCas9, a tool that enables precise gene editing without DNA breakage. Researchers also developed BEAT, a computational program to quantify base editing outcomes. Additionally, the journal reported on identifying genetic vulnerabilities in cancer cells via CRISPR-Cas9.
Researchers developed a new gene editing system that simultaneously suppresses proteins inhibiting the immune system in lymphoma cells and activates cytotoxic T lymphocytes. The technology, based on improved CRISPR gene editing, shows promise for treating various diseases including cancer, autoimmune, and inflammatory conditions.
A novel lipid nanoparticle delivery system has been developed to deliver CRISPR/Cas9 gene editing tools into liver cells with up to 90% efficiency. This improvement has the potential to overcome technical hurdles for clinical applications, including treatment of hyperlipidemia and various diseases.
A CWRU team will provide regulatory guidelines for non-traditional gene editing experiments, aiming to protect the public while encouraging creativity and innovation. The researchers will explore options such as licensure requirements, experiment reporting, and equipment restrictions.
A new study found that people in the US are wary of using CRISPR-based genetic engineering to achieve wildlife conservation goals. The research, conducted by a University of Central Florida researcher, suggests that the public perceives the risks of gene editing as outweighing the benefits.
Researchers from CSHL discovered a cryptic mutation in tomatoes that had unexpected effects on growth and yield. By understanding the interaction between this mutation and another gene, they found that duplicating the mutated gene restored its function, providing a solution to agricultural production issues.
A commentary by Chinese experts critiques the first reported instance of germline gene editing in humans, arguing that it was misconceived due to outdated assumptions about HIV infection. The authors recommend strict laws and regulations to oversee future human germline editing experiments.
A breakthrough CRISPR gene-editing tool allows for the simultaneous execution of multiple edits in DNA extracted from human cells. This technology, developed by the Gene Editing Institute and licensed to NovellusDx, can rapidly reproduce an individual patient's cancer tumor genetic features and identify driver mutations.
The CRISPR Journal publishes research on gene editing technologies, including base editors that enable precise base substitutions without DNA cleavage. A new method for multiplex site-directed mutagenesis also offers great promise for studying gene function.
Using CRISPR gene editing, researchers have successfully treated lethal lung diseases in mice by introducing genetic changes during fetal development. The study shows promise for developing new therapies for congenital lung diseases, such as surfactant protein deficiency and cystic fibrosis.
Researchers used CRISPR to edit genes in mice, targeting cells that line the airways of the lungs. The study demonstrated promising results for developing new therapies for congenital lung diseases such as cystic fibrosis and surfactant protein deficiency.
A new approach to gene editing has been developed by scientists at UMass Medical School, allowing for the correction of microduplications associated with 143 different diseases. The strategy uses CRISPR/Cas9 and harnesses the homology-directed repair pathway to remove duplicated sequences and restore functional genes.
Researchers have developed a gene editing approach that corrects over 80% of patient cells, targeting the COL7A1 gene mutation responsible for recessive dystrophic epidermolysis bullosa. The technique shows promise for treating this rare disease with high efficiency and safety.
Purdue University researchers have developed a new gene editing technology that can modify DNA anywhere in the genome, potentially treating diseases like cancer and improving crop production. The new protein, Argonaute from Natronobacterium gregoryi, enables precise edits with increased accessibility
Researchers have developed a more precise gene-editing technique to reduce DNA breaks and prevent cancer in stem cells. This breakthrough could improve the safety of CRISPR treatments for inherited blood disorders.
Researchers at Duke University found that a single CRISPR treatment can safely correct genetic disease for over a year, despite immune responses. The study suggests approaches to address potential challenges and potentially deliver the therapy to infants or restrict Cas9 expression.
A research team has identified and overcome a barrier in CRISPR gene editing that may lay the foundation for sustained treatments using the technique. By increasing the quantity of 'flags' in CRISPR, they were able to extend the effectiveness of treatment from three months to 18 months in mice with Duchenne muscular dystrophy.
A new study published in Molecular Therapy Oncolytics found that combining chemotherapy with CRISPR-Cas9 can stop tumor growth and reduce existing tumor volume in both tissue culture and mouse models. The CRISPR application disables the NRF2 gene, which helps lung cancer tumors develop resistance to chemotherapies.
Researchers have developed a deeper understanding of the CRISPR-Cas12a mechanism, enabling fine-tuning of the gene-editing process. By mapping the molecular structure and sequence of events, scientists can optimize the technology to achieve desired effects while minimizing side effects.
A new machine learning predictive tool, FORECasT, enables scientists to predict the exact mutations resulting from CRISPR-Cas9 gene editing, saving time and resources. The tool was developed using a massive dataset of 40,000 DNA sequences and analysis of over 1 billion DNA sequences.
Scientists at Newcastle University have identified a gene editing method to stop kidney damage in patients with Joubert syndrome, a genetic condition causing brain and kidney issues. The 'exon-skipping' technique may lead to personalized therapies for inherited kidney diseases.
A new paper urges creation of a coordinating global body to assure neutral evaluation of gene editing's benefits and risks. The technologies have breathtaking promise but also profound concerns about unintended consequences, requiring careful review and inclusion of local communities.
Researchers have found that CRISPR-Cas9 generates a strong immune response in humans, with T cells reacting to the Cas9 protein in almost all healthy subjects. However, the study also highlights the need for new solutions to prevent dangerous immune reactions, particularly for genetic diseases requiring in vivo modifications.
UCSB researchers have developed a highly efficient genome editing method that offers complete spatiotemporal control, allowing users to target specific cells or regions within the cell. This approach enables precise and transient gene editing with minimal long-term effects on DNA.
New technology enhances CRISPR-Cas9 gene editing efficiency in mosquitoes, allowing for easier manipulation of gene expression in a wide range of species. This breakthrough enables control of vector-borne diseases, elimination of agricultural insect pests, and potentially gene therapy for human and animal health.
Researchers found that Cas12a is a more choosier enzyme than Cas9 due to its binding mechanism, making it less likely to edit the wrong part of the genome. This discovery could lead to improved gene editing in plants and animals with increased safety for human applications.
A majority of Americans support using gene editing to treat serious diseases in babies, but are less accepting of its use to boost intelligence or develop gene editing technology. The survey also found that people with high science knowledge and lower religious commitment tend to view gene editing more positively.
The publication demonstrates the company's technology induces efficient and precise in vivo gene editing using homologous recombination, a natural DNA correction pathway. This early academic research translated into a scalable process for genetic medicines development.
Researchers used a peptide nucleic acid-based gene editing technique to successfully cure a genetic condition in mice. The treatment corrected 6% of mutations and caused dramatic improvements in symptoms, suggesting a promising new approach for treating genetic disorders during early stages of development.
The CRISPR Journal announces its third issue with novel techniques for long DNA delivery, correction of recessive genetic defects using endogenous repair, base editing quantification software, leveling the CRISPR playing field through accessible plasmid repositories, and insights into CRISPR's future by Editor-in-Chief Rodolphe Barrangou
Researchers at the University of Bristol have developed a method to enhance red blood cell transfusion compatibility using CRISPR-Cas9 gene editing. By altering specific blood group genes, they can prevent immune reactions in patients with rare blood types or those requiring frequent transfusions.
The CRISPR Journal publishes new research on cell-free CRISPR systems, which enable the study of gene editing mechanisms in a defined manner. Additionally, a universal CRISPR activity model called TUSCAN has been developed to predict CRISPR-Cas9 activity and genome-wide screening tasks.
Scientists at Christiana Care's Gene Editing Institute have developed a breakthrough CRISPR tool that can edit DNA in a test tube, allowing for precise genetic mutations to be replicated from human tumor samples. This technology has the potential to accelerate personalized cancer care by enabling rapid diagnosis and treatment.
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.