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.
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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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateAug 22, 2019
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.
SourceNational Research Council of Science & Technology·JournalBiomaterials·DateAug 20, 2019
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.
SourceTufts University·JournalAdvanced Materials·DateJul 12, 2019
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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.
SourceUniversity of Central Florida·JournalConservation Biology·DateJun 26, 2019
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.
SourceCold Spring Harbor Laboratory·JournalNature Plants·DateMay 6, 2019
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.
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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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateApr 18, 2019
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.
SourceChildren's Hospital of Philadelphia·JournalScience Translational Medicine·DateApr 17, 2019
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.
SourceUniversity of Pennsylvania School of Medicine·JournalScience Translational Medicine·DateApr 17, 2019
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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.
SourceUMass Chan Medical School·JournalNature·DateApr 10, 2019
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.
SourceUniversidad Carlos III de Madrid·JournalMolecular Therapy·DateApr 8, 2019
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.
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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.
SourceDuke University·JournalNature Medicine·DateFeb 18, 2019
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.
SourceUniversity of Missouri-Columbia·JournalJCI Insight·DateJan 8, 2019
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.
SourceBurness·JournalMolecular Therapy — Oncolytics·DateDec 17, 2018
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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.
SourceUniversity of Copenhagen - The Faculty of Health and Medical Sciences·JournalCell·DateNov 29, 2018
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.
SourceWellcome Trust Sanger Institute·JournalNature Biotechnology·DateNov 27, 2018
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.
SourceNewcastle University·JournalProceedings of the National Academy of Sciences·DateNov 16, 2018
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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.
SourceYale School of the Environment·JournalScience·DateNov 1, 2018
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.
SourceCharité - Universitätsmedizin Berlin·JournalNature Medicine·DateOct 30, 2018
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.
SourceUniversity of California - Santa Barbara·JournalSmall·DateAug 21, 2018
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.
SourcePenn State·JournalNature Communications·DateAug 9, 2018
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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.
SourceUniversity of Texas at Austin·JournalMolecular Cell·DateAug 2, 2018
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.
SourceHomology Medicines·JournalProceedings of the National Academy of Sciences·DateJul 16, 2018
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.
SourceCarnegie Mellon University·JournalNature Communications·DateJul 9, 2018
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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
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateJun 18, 2018
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.
SourceUniversity of Bristol·JournalEMBO Molecular Medicine·DateApr 27, 2018
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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateApr 19, 2018
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.
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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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateApr 9, 2018
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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateFeb 15, 2018
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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalThe CRISPR Journal·DateFeb 15, 2018
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.
SourceBoston Children's Hospital·JournalProceedings of the National Academy of Sciences·DateDec 11, 2017
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.
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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.
SourceUniversity of California - Santa Barbara·DateJul 20, 2017
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.
SourceAmerican Association for the Advancement of Science (AAAS)·JournalScience Advances·DateJul 12, 2017
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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.
SourceUniversity of California - Berkeley·JournalScience Advances·DateJul 12, 2017
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.
SourceScripps Research Institute·JournalNature Chemical Biology·DateJun 19, 2017
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.
SourceEmory Health Sciences·JournalJournal of Clinical Investigation·DateJun 19, 2017
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.
SourceMichigan State University·JournalHuman Molecular Genetics·DateMay 1, 2017
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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.
SourceSAGE·JournalJournal of the Royal Society of Medicine·DateApr 26, 2017
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.
SourceInstitute for Basic Science·JournalNature Biotechnology·DateApr 10, 2017
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.
SourceMary Ann Liebert, Inc./Genetic Engineering News·JournalHuman Gene Therapy·DateMar 20, 2017
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.
SourceCell Press·JournalTrends in Plant Science·DateMar 2, 2017
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.
SourceInstitute for Basic Science·JournalNature Biotechnology·DateFeb 27, 2017
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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.
SourceUniversity of Michigan·JournalProceedings of the National Academy of Sciences·DateOct 31, 2016
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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.
SourceCarnegie Mellon University·JournalNature Communications·DateOct 26, 2016
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.
SourceYale University·JournalNature Communications·DateOct 26, 2016
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.
SourceSt. Jude Children's Research Hospital·JournalNature Medicine·DateAug 15, 2016