Therapeutic Gene Editing Safety and Specificity
نویسندگان
چکیده
منابع مشابه
Delivery and Specificity of CRISPR-Cas9 Genome Editing Technologies for Human Gene Therapy.
Genome editing using the clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR associated 9 (Cas9) technology is revolutionizing the study of gene function and likely will give rise to an entire new class of therapeutics for a wide range of diseases. Achieving this goal requires not only characterization of the technology for efficacy and specificity but also optimization of...
متن کاملEthics and germline gene editing.
T he current kerfuffle around the use of CRISPR/Cas9 and other gene editing technologies in human germline research is the latest in a series of related controversies at the intersection of science, medicine, and ethics [1]. Soon after a prominent ad hoc group of scientists called for a moratorium on clinical applications of germline gene editing [2], a research group from China published an ar...
متن کاملGene Editing Versus Morphants
Kok et al. conclude that results with Morpholino oligos, which are not recapitulated by natural mutations or gene editing mutations, must be due to off-target effects and, when morphants and mutants differ, they contend that antisense oligo studies should be disregarded. Morpholinos and gene editing mutagenesis are very different technologies and, even if both work perfectly, may yield differen...
متن کاملGenome editing at the crossroads of delivery, specificity, and fidelity.
Genome editing (GE) entails the modification of specific genomic sequences in living cells for the purpose of determining, changing, or expanding their function(s). Typically, GE occurs after delivering sequence-specific designer nucleases (e.g., ZFNs, TALENs, and CRISPR/Cas9) and donor DNA constructs into target cells. These designer nucleases can generate gene knockouts or gene knock-ins when...
متن کاملTherapeutic gene editing in CD34+ hematopoietic progenitors from Fanconi anemia patients
Gene targeting constitutes a new step in the development of gene therapy for inherited diseases. Although previous studies have shown the feasibility of editing fibroblasts from Fanconi anemia (FA) patients, here we aimed at conducting therapeutic gene editing in clinically relevant cells, such as hematopoietic stem cells (HSCs). In our first experiments, we showed that zinc finger nuclease (ZF...
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ژورنال
عنوان ژورنال: Hematology/Oncology Clinics of North America
سال: 2017
ISSN: 0889-8588
DOI: 10.1016/j.hoc.2017.05.002