Literature DB >> 32949496

Dual Purpose Vectors for Rare Neurological Diseases.

Brian W Bigger1.   

Abstract

Entities:  

Year:  2020        PMID： 32949496      PMCID： PMC7545005          DOI： 10.1016/j.ymthe.2020.09.017

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


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  4 in total

1.  Pronounced Therapeutic Benefit of a Single Bidirectional AAV Vector Administered Systemically in Sandhoff Mice.

Authors:  Hannah G Lahey; Chelsea J Webber; Diane Golebiowski; Cassandra M Izzo; Erin Horn; Toloo Taghian; Paola Rodriguez; Ana Rita Batista; Lauren E Ellis; Misako Hwang; Douglas R Martin; Heather Gray-Edwards; Miguel Sena-Esteves
Journal:  Mol Ther       Date:  2020-06-19       Impact factor: 11.454

2.  Adeno-associated virus-mediated expression of β-hexosaminidase prevents neuronal loss in the Sandhoff mouse brain.

Authors:  Timothy J Sargeant; Susan Wang; Josephine Bradley; Nicolas J C Smith; Animesh A Raha; Rosamund McNair; Robin J Ziegler; Seng H Cheng; Timothy M Cox; Maria Begoña Cachón-González
Journal:  Hum Mol Genet       Date:  2011-08-18       Impact factor: 6.150

3.  Gene transfer corrects acute GM2 gangliosidosis--potential therapeutic contribution of perivascular enzyme flow.

Authors:  M Begoña Cachón-González; Susan Z Wang; Rosamund McNair; Josephine Bradley; David Lunn; Robin Ziegler; Seng H Cheng; Timothy M Cox
Journal:  Mol Ther       Date:  2012-03-27       Impact factor: 11.454

4.  Novel Vector Design and Hexosaminidase Variant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease.

Authors:  Subha Karumuthil-Melethil; Sahana Nagabhushan Kalburgi; Patrick Thompson; Michael Tropak; Michael D Kaytor; John G Keimel; Brian L Mark; Don Mahuran; Jagdeep S Walia; Steven J Gray
Journal:  Hum Gene Ther       Date:  2016-07       Impact factor: 5.695
  4 in total
  1 in total

1.  Letter to the Editor.

Authors:  Brian L Mark; Don Mahuran
Journal:  Mol Ther       Date:  2020-12-11       Impact factor: 11.454
  1 in total

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