Editors' Note Peri, Yoram; Scham, Paul L
Israel studies review,
09/2021, Letnik:
36, Številka:
2
Journal Article
Recenzirano
In the spring of 2011, the two of us took over the editorship of the newly renamed, and somewhat reshaped, official journal of the Association for Israel Studies. The former Israel Studies Forum thus ...became Israel Studies Review. The current issue is the last that we will be editing, after 25 issues comprising over 200 articles written by almost as many different authors, some of whom were chosen by more than a dozen guest editors who produced our special issues. About two hundred colleagues wrote book reviews and review essays, and many more have served as peer reviewers of articles submitted to us for publication.
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and ...cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is-or will soon be-in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.
Genome editing (GE) has revolutionized biological research through the new ability to precisely edit the genomes of living organisms. In recent years, various GE tools have been explored for editing ...simple and complex genomes. The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has widely been used in GE due to its high efficiency, ease of use, and accuracy. It can be used to add desirable and remove undesirable alleles simultaneously in a single event. Here, we discuss various applications of CRISPR/Cas9 in a range of important crops, compare it with other GE tools, and review its mechanism, limitations, and future possibilities. Various newly emerging CRISPR/Cas systems, including base editing (BE), xCas9, and Cas12a (Cpf1), are also considered.
Genome editing (GE) has modernized the biological world by providing a means to edit genomes of living organisms, including humans, plants, animals, and microbes.CRISPR/Cas9 is an RNA guided endonuclease targeting the DNA.CRISPR/Cas9 has high efficiency, accuracy, and ease of use for GE.Until now, the CRISPR/Cas9 system has been the best choice for GE, but despite its extensive use and applications, there are still some limitations to its more widespread application.Newly emerging CRISPR/Cas systems (i.e., spCas9-NG, base editing, xCas9, Cpf1, Cas13, Cas14) are now being used for GE.Base editing (BE) introduces precise and reproducible nucleotide changes at genomic targets without requiring donor DNA templates, double-stranded breaks (DSBs), or dependency on homology-directed repair (HDR) and nonhomologous end-joining (NHEJ).
This edited collection examines the ethical, legal, social and policy implications of genome editing technologies. Moreover, it offers a broad spectrum of timely legal analysis related to bringing ...genome editing to the market and making it available to patients, including addressing genome editing technology regulation through procedures for regulatory approval, patent law and competition law. In twelve chapters, this volume offers persuasive arguments for justifying transformative regulatory interventions regarding human genome editing, as well as the various legal venues for introducing necessary or desirable changes needed to create an environment for realizing the potential of genome editing technology for the benefit of patients and society.
Human Germline Genome Editing Ormond, Kelly E.; Mortlock, Douglas P.; Scholes, Derek T. ...
American journal of human genetics,
08/2017, Letnik:
101, Številka:
2
Journal Article
Recenzirano
Odprti dostop
With CRISPR/Cas9 and other genome-editing technologies, successful somatic and germline genome editing are becoming feasible. To respond, an American Society of Human Genetics (ASHG) workgroup ...developed this position statement, which was approved by the ASHG Board in March 2017. The workgroup included representatives from the UK Association of Genetic Nurses and Counsellors, Canadian Association of Genetic Counsellors, International Genetic Epidemiology Society, and US National Society of Genetic Counselors. These groups, as well as the American Society for Reproductive Medicine, Asia Pacific Society of Human Genetics, British Society for Genetic Medicine, Human Genetics Society of Australasia, Professional Society of Genetic Counselors in Asia, and Southern African Society for Human Genetics, endorsed the final statement. The statement includes the following positions. (1) At this time, given the nature and number of unanswered scientific, ethical, and policy questions, it is inappropriate to perform germline gene editing that culminates in human pregnancy. (2) Currently, there is no reason to prohibit in vitro germline genome editing on human embryos and gametes, with appropriate oversight and consent from donors, to facilitate research on the possible future clinical applications of gene editing. There should be no prohibition on making public funds available to support this research. (3) Future clinical application of human germline genome editing should not proceed unless, at a minimum, there is (a) a compelling medical rationale, (b) an evidence base that supports its clinical use, (c) an ethical justification, and (d) a transparent public process to solicit and incorporate stakeholder input.
Most genetic variants that contribute to disease
are challenging to correct efficiently and without excess byproducts
. Here we describe prime editing, a versatile and precise genome editing method ...that directly writes new genetic information into a specified DNA site using a catalytically impaired Cas9 endonuclease fused to an engineered reverse transcriptase, programmed with a prime editing guide RNA (pegRNA) that both specifies the target site and encodes the desired edit. We performed more than 175 edits in human cells, including targeted insertions, deletions, and all 12 types of point mutation, without requiring double-strand breaks or donor DNA templates. We used prime editing in human cells to correct, efficiently and with few byproducts, the primary genetic causes of sickle cell disease (requiring a transversion in HBB) and Tay-Sachs disease (requiring a deletion in HEXA); to install a protective transversion in PRNP; and to insert various tags and epitopes precisely into target loci. Four human cell lines and primary post-mitotic mouse cortical neurons support prime editing with varying efficiencies. Prime editing shows higher or similar efficiency and fewer byproducts than homology-directed repair, has complementary strengths and weaknesses compared to base editing, and induces much lower off-target editing than Cas9 nuclease at known Cas9 off-target sites. Prime editing substantially expands the scope and capabilities of genome editing, and in principle could correct up to 89% of known genetic variants associated with human diseases.
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