Literature DB >> 27982043

Gene therapy: Gene-editing therapy for neurological disease.

Moira A McMahon1, Don W Cleveland1.   

Abstract

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Year:  2016        PMID： 27982043     DOI： 10.1038/nrneurol.2016.190

Source DB:  PubMed          Journal:  Nat Rev Neurol        ISSN： 1759-4758            Impact factor:   42.937


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  10 in total

1.  Synthetic CRISPR RNA-Cas9-guided genome editing in human cells.

Authors:  Meghdad Rahdar; Moira A McMahon; Thazha P Prakash; Eric E Swayze; C Frank Bennett; Don W Cleveland
Journal:  Proc Natl Acad Sci U S A       Date:  2015-11-16       Impact factor: 11.205

Review 2.  Molecular mechanisms of fragile X syndrome: a twenty-year perspective.

Authors:  Michael R Santoro; Steven M Bray; Stephen T Warren
Journal:  Annu Rev Pathol       Date:  2011-10-10       Impact factor: 23.472

3.  Antisense oligonucleotide therapy for neurodegenerative disease.

Authors:  Richard A Smith; Timothy M Miller; Koji Yamanaka; Brett P Monia; Thomas P Condon; Gene Hung; Christian S Lobsiger; Chris M Ward; Melissa McAlonis-Downes; Hongbing Wei; Ed V Wancewicz; C Frank Bennett; Don W Cleveland
Journal:  J Clin Invest       Date:  2006-07-27       Impact factor: 14.808

4.  Therapeutic AAV9-mediated suppression of mutant SOD1 slows disease progression and extends survival in models of inherited ALS.

Authors:  Kevin D Foust; Desirée L Salazar; Shibi Likhite; Laura Ferraiuolo; Dara Ditsworth; Hristelina Ilieva; Kathrin Meyer; Leah Schmelzer; Lyndsey Braun; Don W Cleveland; Brian K Kaspar
Journal:  Mol Ther       Date:  2013-09-06       Impact factor: 11.454

5.  Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR/Cas9.

Authors:  Jun Wan Shin; Kyung-Hee Kim; Michael J Chao; Ranjit S Atwal; Tammy Gillis; Marcy E MacDonald; James F Gusella; Jong-Min Lee
Journal:  Hum Mol Genet       Date:  2016-10-15       Impact factor: 6.150

Review 6.  Therapeutic genome editing: prospects and challenges.

Authors:  David Benjamin Turitz Cox; Randall Jeffrey Platt; Feng Zhang
Journal:  Nat Med       Date:  2015-02       Impact factor: 53.440

7.  A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity.

Authors:  Martin Jinek; Krzysztof Chylinski; Ines Fonfara; Michael Hauer; Jennifer A Doudna; Emmanuelle Charpentier
Journal:  Science       Date:  2012-06-28       Impact factor: 47.728

8.  Multiplex genome engineering using CRISPR/Cas systems.

Authors:  Le Cong; F Ann Ran; David Cox; Shuailiang Lin; Robert Barretto; Naomi Habib; Patrick D Hsu; Xuebing Wu; Wenyan Jiang; Luciano A Marraffini; Feng Zhang
Journal:  Science       Date:  2013-01-03       Impact factor: 47.728

9.  Reversion of FMR1 Methylation and Silencing by Editing the Triplet Repeats in Fragile X iPSC-Derived Neurons.

Authors:  Chul-Yong Park; Tomer Halevy; Dongjin R Lee; Jin Jea Sung; Jae Souk Lee; Ofra Yanuka; Nissim Benvenisty; Dong-Wook Kim
Journal:  Cell Rep       Date:  2015-10-01       Impact factor: 9.423

10.  Reactivation of FMR1 by CRISPR/Cas9-Mediated Deletion of the Expanded CGG-Repeat of the Fragile X Chromosome.

Authors:  Nina Xie; He Gong; Joshua A Suhl; Pankaj Chopra; Tao Wang; Stephen T Warren
Journal:  PLoS One       Date:  2016-10-21       Impact factor: 3.240
  10 in total
  9 in total

1.  Chemically Modified Cpf1-CRISPR RNAs Mediate Efficient Genome Editing in Mammalian Cells.

Authors:  Moira A McMahon; Thazha P Prakash; Don W Cleveland; C Frank Bennett; Meghdad Rahdar
Journal:  Mol Ther       Date:  2018-03-06       Impact factor: 11.454

2.  Nanoparticle delivery of CRISPR into the brain rescues a mouse model of fragile X syndrome from exaggerated repetitive behaviours.

Authors:  Bumwhee Lee; Kunwoo Lee; Shree Panda; Rodrigo Gonzales-Rojas; Anthony Chong; Vladislav Bugay; Hyo Min Park; Robert Brenner; Niren Murthy; Hye Young Lee
Journal:  Nat Biomed Eng       Date:  2018-06-25       Impact factor: 25.671

Review 3.  Close encounters: Moving along bumps, breaks, and bubbles on expanded trinucleotide tracts.

Authors:  Aris A Polyzos; Cynthia T McMurray
Journal:  DNA Repair (Amst)       Date:  2017-06-09

Review 4.  Designer protein disaggregases to counter neurodegenerative disease.

Authors:  James Shorter
Journal:  Curr Opin Genet Dev       Date:  2017-02-14       Impact factor: 5.578

Review 5.  Active genetics comes alive: Exploring the broad applications of CRISPR-based selfish genetic elements (or gene-drives): Exploring the broad applications of CRISPR-based selfish genetic elements (or gene-drives).

Authors:  Valentino M Gantz; Ethan Bier
Journal:  Bioessays       Date:  2022-06-09       Impact factor: 4.653

Review 6.  Advances in nanotechnology-based strategies for the treatments of amyotrophic lateral sclerosis.

Authors:  G Y Wang; S L Rayner; R Chung; B Y Shi; X J Liang
Journal:  Mater Today Bio       Date:  2020-05-04

Review 7.  The cell-based approach in neurosurgery: ongoing trends and future perspectives.

Authors:  Sabino Luzzi; Alberto Maria Crovace; Mattia Del Maestro; Alice Giotta Lucifero; Samer K Elbabaa; Benedetta Cinque; Paola Palumbo; Francesca Lombardi; Annamaria Cimini; Maria Grazia Cifone; Antonio Crovace; Renato Galzio
Journal:  Heliyon       Date:  2019-11-26

Review 8.  Unraveling of Central Nervous System Disease Mechanisms Using CRISPR Genome Manipulation.

Authors:  Aino Vesikansa
Journal:  J Cent Nerv Syst Dis       Date:  2018-07-10

9.  CRISPR/Cas9 editing of APP C-terminus attenuates β-cleavage and promotes α-cleavage.

Authors:  Jichao Sun; Jared Carlson-Stevermer; Utpal Das; Minjie Shen; Marion Delenclos; Amanda M Snead; So Yeon Koo; Lina Wang; Dianhua Qiao; Jonathan Loi; Andrew J Petersen; Michael Stockton; Anita Bhattacharyya; Mathew V Jones; Xinyu Zhao; Pamela J McLean; Andrew A Sproul; Krishanu Saha; Subhojit Roy
Journal:  Nat Commun       Date:  2019-01-03       Impact factor: 14.919
  9 in total

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