Warning: Undefined array key "mm" in /www/wwwroot/www.ai-bt.com/si.php on line 10 Deprecated: trim(): Passing null to parameter #1 ($string) of type string is deprecated in /www/wwwroot/www.ai-bt.com/si.php on line 10 Pompe disease: how to solve many problems with one solution.

Literature DB >> 30211201

Pompe disease: how to solve many problems with one solution.

Rosa Puertollano¹, Nina Raben¹.

Abstract

Year: 2018 PMID： 30211201 PMCID： PMC6123209 DOI： 10.21037/atm.2018.06.52

Source DB: PubMed Journal: Ann Transl Med ISSN： 2305-5839

× No keyword cloud information.

24 in total

1. Therapeutic Benefit of Autophagy Modulation in Pompe Disease.

Authors: Jeong-A Lim; Baodong Sun; Rosa Puertollano; Nina Raben
Journal: Mol Ther Date: 2018-05-03 Impact factor: 11.454

2. Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.

Authors: Principal Investigators Barry J Byrne; Shelley Collins; Coinvestigators Cathryn Mah; Barbara Smith; Thomas Conlon; Subinvestigators Danny Martin; Manuela Corti; Brian Cleaver; Saleem Islam; Lee Ann Lawson
Journal: Hum Gene Ther Clin Dev Date: 2014-09 Impact factor: 5.032

3. Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models.

Authors: Randy J Chandler; Mark S Sands; Charles P Venditti
Journal: Hum Gene Ther Date: 2017-04 Impact factor: 5.695

Review 4. Synonymous but not the same: the causes and consequences of codon bias.

Authors: Joshua B Plotkin; Grzegorz Kudla
Journal: Nat Rev Genet Date: 2010-11-23 Impact factor: 53.242

5. Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance.

Authors: Baodong Sun; Andrew Bird; Sarah P Young; Priya S Kishnani; Y-T Chen; Dwight D Koeberl
Journal: Am J Hum Genet Date: 2007-09-21 Impact factor: 11.025

6. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice.

Authors: Robin J Ziegler; Scott D Bercury; Jonathan Fidler; Michael A Zhao; Joseph Foley; Tatyana V Taksir; Susan Ryan; Bradley L Hodges; Ronald K Scheule; Lamya S Shihabuddin; Seng H Cheng
Journal: Hum Gene Ther Date: 2008-06 Impact factor: 5.695

7. Early detection of Pompe disease by newborn screening is feasible: results from the Taiwan screening program.

Authors: Yin-Hsiu Chien; Shu-Chuan Chiang; Xiaokui Kate Zhang; Joan Keutzer; Ni-Chung Lee; Ai-Chu Huang; Chun-An Chen; Mei-Hwan Wu; Pei-Hsin Huang; Fu-Jen Tsai; Yuan-Tsong Chen; Wuh-Liang Hwu
Journal: Pediatrics Date: 2008-06-02 Impact factor: 7.124

8. The emerging phenotype of long-term survivors with infantile Pompe disease.

Authors: Sean N Prater; Suhrad G Banugaria; Stephanie M DeArmey; Eleanor G Botha; Erin M Stege; Laura E Case; Harrison N Jones; Chanika Phornphutkul; Raymond Y Wang; Sarah P Young; Priya S Kishnani
Journal: Genet Med Date: 2012-04-26 Impact factor: 8.822

9. The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease.

Authors: Suhrad G Banugaria; Sean N Prater; Yiu-Ki Ng; Joyce A Kobori; Richard S Finkel; Roger L Ladda; Yuan-Tsong Chen; Amy S Rosenberg; Priya S Kishnani
Journal: Genet Med Date: 2011-08 Impact factor: 8.822