Literature DB >> 28341564

Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases.

Dominic J Wells1.   

Abstract

Mesh:

Year:  2017        PMID: 28341564      PMCID: PMC5383641          DOI: 10.1016/j.ymthe.2017.03.006

Source DB:  PubMed          Journal:  Mol Ther        ISSN: 1525-0016            Impact factor:   11.454


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  13 in total

1.  Dystrophin immunity in Duchenne's muscular dystrophy.

Authors:  Jerry R Mendell; Katherine Campbell; Louise Rodino-Klapac; Zarife Sahenk; Chris Shilling; Sarah Lewis; Dawn Bowles; Steven Gray; Chengwen Li; Gloria Galloway; Vinod Malik; Brian Coley; K Reed Clark; Juan Li; Xiao Xiao; Jade Samulski; Scott W McPhee; R Jude Samulski; Christopher M Walker
Journal:  N Engl J Med       Date:  2010-10-07       Impact factor: 91.245

2.  Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

Authors:  Jerry R Mendell; Louise R Rodino-Klapac; Xiomara Q Rosales; Brian D Coley; Gloria Galloway; Sarah Lewis; Vinod Malik; Chris Shilling; Barry J Byrne; Thomas Conlon; Katherine J Campbell; William G Bremer; Laura E Taylor; Kevin M Flanigan; Julie M Gastier-Foster; Caroline Astbury; Janaiah Kota; Zarife Sahenk; Christopher M Walker; K Reed Clark
Journal:  Ann Neurol       Date:  2010-11       Impact factor: 10.422

3.  Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy.

Authors:  Martin K Childers; Romain Joubert; Karine Poulard; Christelle Moal; Robert W Grange; Jonathan A Doering; Michael W Lawlor; Branden E Rider; Thibaud Jamet; Nathalie Danièle; Samia Martin; Christel Rivière; Thomas Soker; Caroline Hammer; Laetitia Van Wittenberghe; Mandy Lockard; Xuan Guan; Melissa Goddard; Erin Mitchell; Jane Barber; J Koudy Williams; David L Mack; Mark E Furth; Alban Vignaud; Carole Masurier; Fulvio Mavilio; Philippe Moullier; Alan H Beggs; Anna Buj-Bello
Journal:  Sci Transl Med       Date:  2014-01-22       Impact factor: 17.956

4.  A phase 1/2a follistatin gene therapy trial for becker muscular dystrophy.

Authors:  Jerry R Mendell; Zarife Sahenk; Vinod Malik; Ana M Gomez; Kevin M Flanigan; Linda P Lowes; Lindsay N Alfano; Katherine Berry; Eric Meadows; Sarah Lewis; Lyndsey Braun; Kim Shontz; Maria Rouhana; Kelly Reed Clark; Xiomara Q Rosales; Samiah Al-Zaidy; Alessandra Govoni; Louise R Rodino-Klapac; Mark J Hogan; Brian K Kaspar
Journal:  Mol Ther       Date:  2014-10-17       Impact factor: 11.454

5.  Hydrodynamic limb vein injection of adeno-associated virus serotype 8 vector carrying canine myostatin propeptide gene into normal dogs enhances muscle growth.

Authors:  Chunping Qiao; Juan Li; Hui Zheng; Janet Bogan; Jianbin Li; Zhenhua Yuan; Cheng Zhang; Dan Bogan; Joe Kornegay; Xiao Xiao
Journal:  Hum Gene Ther       Date:  2009-01       Impact factor: 5.695

6.  Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus.

Authors:  Yongping Yue; Xiufang Pan; Chady H Hakim; Kasun Kodippili; Keqing Zhang; Jin-Hong Shin; Hsiao T Yang; Thomas McDonald; Dongsheng Duan
Journal:  Hum Mol Genet       Date:  2015-08-11       Impact factor: 6.150

7.  Forelimb treatment in a large cohort of dystrophic dogs supports delivery of a recombinant AAV for exon skipping in Duchenne patients.

Authors:  Caroline Le Guiner; Marie Montus; Laurent Servais; Yan Cherel; Virginie Francois; Jean-Laurent Thibaud; Claire Wary; Béatrice Matot; Thibaut Larcher; Lydie Guigand; Maeva Dutilleul; Claire Domenger; Marine Allais; Maud Beuvin; Amélie Moraux; Johanne Le Duff; Marie Devaux; Nicolas Jaulin; Mickaël Guilbaud; Virginie Latournerie; Philippe Veron; Sylvie Boutin; Christian Leborgne; Diana Desgue; Jack-Yves Deschamps; Sophie Moullec; Yves Fromes; Adeline Vulin; Richard H Smith; Nicolas Laroudie; Frédéric Barnay-Toutain; Christel Rivière; Stéphanie Bucher; Thanh-Hoa Le; Nicolas Delaunay; Mehdi Gasmi; Robert M Kotin; Gisèle Bonne; Oumeya Adjali; Carole Masurier; Jean-Yves Hogrel; Pierre Carlier; Philippe Moullier; Thomas Voit
Journal:  Mol Ther       Date:  2014-08-04       Impact factor: 11.454

8.  Testosterone-induced increase in muscle size in healthy young men is associated with muscle fiber hypertrophy.

Authors:  Indrani Sinha-Hikim; Jorge Artaza; Linda Woodhouse; Nestor Gonzalez-Cadavid; Atam B Singh; Martin I Lee; Thomas W Storer; Richard Casaburi; Ruoquing Shen; Shalender Bhasin
Journal:  Am J Physiol Endocrinol Metab       Date:  2002-07       Impact factor: 4.310

Review 9.  Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.

Authors:  Timothy C Nichols; Margaret H Whitford; Valder R Arruda; Hansell H Stedman; Mark A Kay; Katherine A High
Journal:  Hum Gene Ther Clin Dev       Date:  2015-02-12

10.  Follistatin Gene Therapy Improves Ambulation in Becker Muscular Dystrophy.

Authors:  Samiah A Al-Zaidy; Zarife Sahenk; Louise R Rodino-Klapac; Brian Kaspar; Jerry R Mendell
Journal:  J Neuromuscul Dis       Date:  2015-09-02
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  6 in total

1.  Targeted tPA overexpression in denervated spinal motor neurons promotes stroke recovery in mice.

Authors:  Xinling Gan; Michael Chopp; Hongqi Xin; Fengjie Wang; William Golembieski; Mei Lu; Li He; Zhongwu Liu
Journal:  J Cereb Blood Flow Metab       Date:  2020-01-27       Impact factor: 6.200

2.  Experimental Gene Therapy with Serine-Histogranin and Endomorphin 1 for the Treatment of Chronic Neuropathic Pain.

Authors:  Stanislava Jergova; Catherine E Gordon; Shyam Gajavelli; Jacqueline Sagen
Journal:  Front Mol Neurosci       Date:  2017-12-08       Impact factor: 5.639

Review 3.  Neutralizing Anti-Hemagglutinin Monoclonal Antibodies Induced by Gene-Based Transfer Have Prophylactic and Therapeutic Effects on Influenza Virus Infection.

Authors:  Tatsuya Yamazaki; Joe Chiba; Sachiko Akashi-Takamura
Journal:  Vaccines (Basel)       Date:  2018-06-26

4.  Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality.

Authors:  Marti Cabanes-Creus; Samantha L Ginn; Anais K Amaya; Sophia H Y Liao; Adrian Westhaus; Claus V Hallwirth; Patrick Wilmott; Jason Ward; Kimberley L Dilworth; Giorgia Santilli; Arkadiusz Rybicki; Hiroyuki Nakai; Adrian J Thrasher; Adrian C Filip; Ian E Alexander; Leszek Lisowski
Journal:  Mol Ther Methods Clin Dev       Date:  2018-11-01       Impact factor: 6.698

5.  Descending motor circuitry required for NT-3 mediated locomotor recovery after spinal cord injury in mice.

Authors:  Qi Han; Josue D Ordaz; Nai-Kui Liu; Zoe Richardson; Wei Wu; Yongzhi Xia; Wenrui Qu; Ying Wang; Heqiao Dai; Yi Ping Zhang; Christopher B Shields; George M Smith; Xiao-Ming Xu
Journal:  Nat Commun       Date:  2019-12-20       Impact factor: 14.919

6.  Expandable and implantable bioelectronic complex for analyzing and regulating real-time activity of the urinary bladder.

Authors:  Tae-Min Jang; Joong Hoon Lee; Honglei Zhou; Jaesun Joo; Bong Hee Lim; Huanyu Cheng; Soo Hyun Kim; Il-Suk Kang; Kyu-Sung Lee; Eunkyoung Park; Suk-Won Hwang
Journal:  Sci Adv       Date:  2020-11-11       Impact factor: 14.136

  6 in total

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