Genome Editing for CNS Disorders.

Frontiers in Neuroscience
Fábio Duarte, Nicole Déglon

Abstract

Central nervous system (CNS) disorders have a social and economic burden on modern societies, and the development of effective therapies is urgently required. Gene editing may prevent or cure a disease by inducing genetic changes at endogenous loci. Genome editing includes not only the insertion, deletion or replacement of nucleotides, but also the modulation of gene expression and epigenetic editing. Emerging technologies based on ZFs, TALEs, and CRISPR/Cas systems have extended the boundaries of genome manipulation and promoted genome editing approaches to the level of promising strategies for counteracting genetic diseases. The parallel development of efficient delivery systems has also increased our access to the CNS. In this review, we describe the various tools available for genome editing and summarize in vivo preclinical studies of CNS genome editing, whilst considering current limitations and alternative approaches to overcome some bottlenecks.

References

Dec 24, 1996·Proceedings of the National Academy of Sciences of the United States of America·S S KimJ V Bonventre
Oct 28, 1999·Clinical Neurology and Neurosurgery·L A LaanO F Brouwer
Jun 2, 2001·The New England Journal of Medicine·L P Rowland, N A Shneider
Aug 9, 2001·Journal of Molecular Biology·E S VanameeA K Aggarwal
Jul 9, 2003·Immunological Reviews·Kevin D MillsFrederick W Alt
Mar 26, 2004·Clinical & Experimental Optometry : Journal of the Australian Optometrical Association·Michael Kalloniatis, Erica L Fletcher
Apr 14, 2005·Blood·Carol A Sledz, Bryan R G Williams
Dec 24, 2005·Human Molecular Genetics·Zdenek BergerDavid C Rubinsztein
Jun 3, 2006·Science·Séverine BoilléeDon W Cleveland
Jul 24, 2007·Trends in Cell Biology·Guillermo M Elias, Roger A Nicoll
Jan 19, 2008·The Journal of Physiology·Gül Dölen, Mark F Bear
Feb 5, 2008·Nature Neuroscience·Koji YamanakaDon W Cleveland
Jun 14, 2008·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Michelle GrayX William Yang
Jul 16, 2008·Nature Reviews. Genetics·Keith W Caldecott
Dec 11, 2008·Molecular Therapy : the Journal of the American Society of Gene Therapy·Hilda Petrs-SilvaWilliam W Hauswirth
Apr 30, 2009·Annual Review of Phytopathology·Jens Boch, Ulla Bonas
Aug 19, 2010·Nature Reviews. Genetics·Fyodor D UrnovPhilip D Gregory
Dec 15, 2010·The Journal of Neuroscience : the Official Journal of the Society for Neuroscience·Josee LaganiereH Steve Zhang
Mar 29, 2011·Nature·Jason ErnstBradley E Bernstein
Jan 31, 2012·Pediatric Clinics of North America·Gabriel Kaplan, James T McCracken
Feb 18, 2012·Nature·Blake WiedenheftJennifer A Doudna
Mar 1, 2012·Methods in Enzymology·Bettina KlugJens Reinhardt
Jun 30, 2012·Science·Martin JinekEmmanuelle Charpentier
Sep 29, 2012·Journal of Neurotrauma·Ishita SiddiqAndrew J Baker
Oct 2, 2012·Molecular Therapy : the Journal of the American Society of Gene Therapy·Seppo Ylä-Herttuala
Oct 12, 2012·Proceedings of the National Academy of Sciences of the United States of America·Mireia Garriga-CanutMark Isalan
Jan 5, 2013·Science·Le CongFeng Zhang
Jan 5, 2013·Science·Prashant MaliGeorge M Church
Apr 9, 2013·Drugs·Philip HairKate McKeage
Jun 19, 2013·Behavioural Brain Research·Antonio M Persico, Valerio Napolioni
Aug 2, 2014·Neuroscience Bulletin·Tian-Lin Cheng, Zilong Qiu
Oct 14, 2014·Cell·Luke A GilbertJonathan S Weissman

❮ Previous
Next ❯

Citations


❮ Previous
Next ❯

Methods Mentioned

BETA
antisense oligonucleotides
SMA
acetylation
transgenic
histone acetylation
GUIDESeq

Clinical Trials Mentioned

NCT03872479

Software Mentioned

SUperNova
GUIDESeq

Related Concepts

Related Feeds

CRISPR (general)

Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). CRISPR-Cas system enables the editing of genes to create or correct mutations. Discover the latest research on CRISPR here.

CRISPR for Genome Editing

Genome editing technologies enable the editing of genes to create or correct mutations. Clustered regularly interspaced short palindromic repeats (CRISPR) are DNA sequences in the genome that are recognized and cleaved by CRISPR-associated proteins (Cas). Here is the latest research on the use of CRISPR-Cas system in gene editing.