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Literature DB >> 31772140

News Feature: Gene therapy successes point to better therapies.

Elie Dolgin.

Abstract

Mesh：

Substances：

Year: 2019 PMID： 31772140 PMCID： PMC6883820 DOI： 10.1073/pnas.1918306116

Source DB: PubMed Journal: Proc Natl Acad Sci U S A ISSN： 0027-8424 Impact factor: 11.205

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15 in total

1. The human centromeric survival motor neuron gene (SMN2) rescues embryonic lethality in Smn(-/-) mice and results in a mouse with spinal muscular atrophy.

Authors: U R Monani; M Sendtner; D D Coovert; D W Parsons; C Andreassi; T T Le; S Jablonka; B Schrank; W Rossoll; W Rossol; T W Prior; G E Morris; A H Burghes
Journal: Hum Mol Genet Date: 2000-02-12 Impact factor: 6.150

2. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons.

Authors: Sandra Duque; Béatrice Joussemet; Christel Riviere; Thibaut Marais; Laurence Dubreil; Anne-Marie Douar; John Fyfe; Philippe Moullier; Marie-Anne Colle; Martine Barkats
Journal: Mol Ther Date: 2009-04-14 Impact factor: 11.454

3. Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy.

Authors: Jerry R Mendell; Samiah Al-Zaidy; Richard Shell; W Dave Arnold; Louise R Rodino-Klapac; Thomas W Prior; Linda Lowes; Lindsay Alfano; Katherine Berry; Kathleen Church; John T Kissel; Sukumar Nagendran; James L'Italien; Douglas M Sproule; Courtney Wells; Jessica A Cardenas; Marjet D Heitzer; Allan Kaspar; Sarah Corcoran; Lyndsey Braun; Shibi Likhite; Carlos Miranda; Kathrin Meyer; K D Foust; Arthur H M Burghes; Brian K Kaspar
Journal: N Engl J Med Date: 2017-11-02 Impact factor: 91.245

4. 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

5. Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.

Authors: Jinkuk Kim; Chunguang Hu; Christelle Moufawad El Achkar; Lauren E Black; Julie Douville; Austin Larson; Mary K Pendergast; Sara F Goldkind; Eunjung A Lee; Ashley Kuniholm; Aubrie Soucy; Jai Vaze; Nandkishore R Belur; Kristina Fredriksen; Iva Stojkovska; Alla Tsytsykova; Myriam Armant; Renata L DiDonato; Jaejoon Choi; Laura Cornelissen; Luis M Pereira; Erika F Augustine; Casie A Genetti; Kira Dies; Brenda Barton; Lucinda Williams; Benjamin D Goodlett; Bobbie L Riley; Amy Pasternak; Emily R Berry; Kelly A Pflock; Stephen Chu; Chantal Reed; Kimberly Tyndall; Pankaj B Agrawal; Alan H Beggs; P Ellen Grant; David K Urion; Richard O Snyder; Susan E Waisbren; Annapurna Poduri; Peter J Park; Al Patterson; Alessandra Biffi; Joseph R Mazzulli; Olaf Bodamer; Charles B Berde; Timothy W Yu
Journal: N Engl J Med Date: 2019-10-09 Impact factor: 91.245

6. Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy.

Authors: Marco A Passini; Jie Bu; Amy M Richards; Cathrine Kinnecom; S Pablo Sardi; Lisa M Stanek; Yimin Hua; Frank Rigo; John Matson; Gene Hung; Edward M Kaye; Lamya S Shihabuddin; Adrian R Krainer; C Frank Bennett; Seng H Cheng
Journal: Sci Transl Med Date: 2011-03-02 Impact factor: 17.956

7. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes.

Authors: Kevin D Foust; Emily Nurre; Chrystal L Montgomery; Anna Hernandez; Curtis M Chan; Brian K Kaspar
Journal: Nat Biotechnol Date: 2008-12-21 Impact factor: 54.908

8. 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

9. Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models.

Authors: Alex McCampbell; Tracy Cole; Amy J Wegener; Giulio S Tomassy; Amy Setnicka; Brandon J Farley; Kathleen M Schoch; Mariah L Hoye; Mark Shabsovich; Linhong Sun; Yi Luo; Mingdi Zhang; Nicole Comfort; Bin Wang; Jessica Amacker; Sai Thankamony; David W Salzman; Merit Cudkowicz; Danielle L Graham; C Frank Bennett; Holly B Kordasiewicz; Eric E Swayze; Timothy M Miller
Journal: J Clin Invest Date: 2018-07-16 Impact factor: 14.808

10. Pharmacology of a central nervous system delivered 2'-O-methoxyethyl-modified survival of motor neuron splicing oligonucleotide in mice and nonhuman primates.

Authors: Frank Rigo; Seung J Chun; Daniel A Norris; Gene Hung; Sam Lee; John Matson; Robert A Fey; Hans Gaus; Yimin Hua; John S Grundy; Adrian R Krainer; Scott P Henry; C Frank Bennett
Journal: J Pharmacol Exp Ther Date: 2014-04-30 Impact factor: 4.030

4 in total

Review 1. Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.

Authors: Paul Boucher; Xiaoxia Cui; David T Curiel
Journal: J Control Release Date: 2020-09-03 Impact factor: 9.776

2. Case Report: Whole-Exome Sequencing With MLPA Revealed Variants in Two Genes in a Patient With Combined Manifestations of Spinal Muscular Atrophy and Duchenne Muscular Dystrophy.

Authors: Yu Xia; Yijie Feng; Lu Xu; Xiaoyang Chen; Feng Gao; Shanshan Mao
Journal: Front Genet Date: 2021-03-10 Impact factor: 4.599

Review 3. RNA-based therapeutics for neurological diseases.

Authors: Karen Anthony
Journal: RNA Biol Date: 2021-12-31 Impact factor: 4.652

Review 4. Emerging Oligonucleotide Therapeutics for Rare Neuromuscular Diseases.

Authors: Yoshitsugu Aoki; Matthew J A Wood
Journal: J Neuromuscul Dis Date: 2021

4 in total