Literature DB >> 33824521

Genetic therapies offer new hope against incurable brain diseases.

Diana Kwon.   

Abstract

Entities:  

Keywords:  Drug discovery; Genetics; Neurodegeneration

Mesh:

Substances:

Year:  2021        PMID: 33824521     DOI: 10.1038/d41586-021-00870-x

Source DB:  PubMed          Journal:  Nature        ISSN: 0028-0836            Impact factor:   49.962


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  5 in total

1.  Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide.

Authors:  M L Stephenson; P C Zamecnik
Journal:  Proc Natl Acad Sci U S A       Date:  1978-01       Impact factor: 11.205

2.  Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.

Authors:  Richard S Finkel; Eugenio Mercuri; Basil T Darras; Anne M Connolly; Nancy L Kuntz; Janbernd Kirschner; Claudia A Chiriboga; Kayoko Saito; Laurent Servais; Eduardo Tizzano; Haluk Topaloglu; Már Tulinius; Jacqueline Montes; Allan M Glanzman; Kathie Bishop; Z John Zhong; Sarah Gheuens; C Frank Bennett; Eugene Schneider; Wildon Farwell; Darryl C De Vivo
Journal:  N Engl J Med       Date:  2017-11-02       Impact factor: 91.245

3.  Antisense masking of an hnRNP A1/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice.

Authors:  Yimin Hua; Timothy A Vickers; Hazeem L Okunola; C Frank Bennett; Adrian R Krainer
Journal:  Am J Hum Genet       Date:  2008-03-27       Impact factor: 11.025

4.  Reversing a model of Parkinson's disease with in situ converted nigral neurons.

Authors:  Hao Qian; Xinjiang Kang; Jing Hu; Dongyang Zhang; Zhengyu Liang; Fan Meng; Xuan Zhang; Yuanchao Xue; Roy Maimon; Steven F Dowdy; Neal K Devaraj; Zhuan Zhou; William C Mobley; Don W Cleveland; Xiang-Dong Fu
Journal:  Nature       Date:  2020-06-24       Impact factor: 49.962

5.  Targeting Huntingtin Expression in Patients with Huntington's Disease.

Authors:  Sarah J Tabrizi; Blair R Leavitt; G Bernhard Landwehrmeyer; Edward J Wild; Carsten Saft; Roger A Barker; Nick F Blair; David Craufurd; Josef Priller; Hugh Rickards; Anne Rosser; Holly B Kordasiewicz; Christian Czech; Eric E Swayze; Daniel A Norris; Tiffany Baumann; Irene Gerlach; Scott A Schobel; Erika Paz; Anne V Smith; C Frank Bennett; Roger M Lane
Journal:  N Engl J Med       Date:  2019-05-06       Impact factor: 91.245

  5 in total

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