DGK and DZHK position paper on genome editing: basic science applications and future perspective

被引：0
作者：
Ralf P. Brandes
Anne Dueck
Stefan Engelhardt
Manuel Kaulich
Christian Kupatt
Maria Teresa De Angelis
Matthias S. Leisegang
Ferdinand le Noble
Alessandra Moretti
Oliver J. Müller
Boris V. Skryabin
Thomas Thum
Wolfgang Wurst
机构：
[1] DZHK-German Center for Cardiovascular Research,Institute for Cardiovascular Physiology
[2] Goethe University,Institut Für Pharmakologie und Toxikologie
[3] Technische Universität München,Institute of Developmental Genetics, Helmholtz Zentrum München Germany
[4] Technische Universität München-Weihenstephan,Institute of Biochemistry II, Medical Faculty
[5] Goethe University Frankfurt,First Medical Department (Cardiology)
[6] University Hospital,Department of Cell and Developmental Biology
[7] Klinikum rechts der Isar,Department of Internal Medicine III
[8] Technische Universität München,Medical Faculty, Core Facility Transgenic Animal and Genetic Engineering Models (TRAM)
[9] ZOO-2 and ITG,Institute of Molecular and Translational Therapeutic Strategies
[10] Karlsruhe Institute of Technology (KIT),Fraunhofer Institute for Toxicology and Experimental Medicine
[11] University of Kiel,DZNE
[12] University of Muenster,undefined
[13] Hannover Medical School,undefined
[14] Fraunhofer Institute,undefined
[15] German Center for Neurodegenerative Diseases,undefined
[16] Site Munich,undefined
[17] Munich Cluster for Systems Neurology (SyNergy),undefined
来源：
Basic Research in Cardiology | 2021年 / 116卷
关键词：
Animal models; CRISPR/Cas; Genome editing; Animal models;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
For a long time, gene editing had been a scientific concept, which was limited to a few applications. With recent developments, following the discovery of TALEN zinc-finger endonucleases and in particular the CRISPR/Cas system, gene editing has become a technique applicable in most laboratories. The current gain- and loss-of function models in basic science are revolutionary as they allow unbiased screens of unprecedented depth and complexity and rapid development of transgenic animals. Modifications of CRISPR/Cas have been developed to precisely interrogate epigenetic regulation or to visualize DNA complexes. Moreover, gene editing as a clinical treatment option is rapidly developing with first trials on the way. This article reviews the most recent progress in the field, covering expert opinions gathered during joint conferences on genome editing of the German Cardiac Society (DGK) and the German Center for Cardiovascular Research (DZHK). Particularly focusing on the translational aspect and the combination of cellular and animal applications, the authors aim to provide direction for the development of the field and the most frequent applications with their problems.
引用
收藏
相关论文
共 1421 条
[1]  
Abudayyeh OO(2017)RNA targeting with CRISPR-Cas13 Nature 550 280-284
[2]  
Gootenberg JS(2018)Large deletions induced by Cas9 cleavage Nature 560 E8-E9
[3]  
Essletzbichler P(2018)The CRISPR tool kit for genome editing and beyond Nat Commun 9 1911-91
[4]  
Han S(2015)A new age in functional genomics using CRISPR/Cas9 in arrayed library screening Front Genet 6 300-157
[5]  
Joung J(2018)Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy Science 362 86-844
[6]  
Belanto JJ(2019)Search-and-replace genome editing without double-strand breaks or donor DNA Nature 576 149-708
[7]  
Verdine V(2020)Genome editing with CRISPR-Cas nucleases, base editors, transposases and prime editors Nat Biotechnol 38 824-153
[8]  
Cox DBT(2012)The AAV vector toolkit: poised at the clinical crossroads Mol Ther 20 699-1897
[9]  
Kellner MJ(2014)Highly efficient CRISPR/Cas9-mediated knock-in in zebrafish by homology-independent DNA repair Genome Res 24 142-516
[10]  
Regev A(2015)Long-term effect of gene therapy on Leber's congenital amaurosis N Engl J Med 372 1887-3175
12345678910