Literature DB >> 33321097

Letter to the Editor.

Brian L Mark1, Don Mahuran2.   

Abstract

Entities:  

Year:  2020        PMID： 33321097      PMCID： PMC7791075          DOI： 10.1016/j.ymthe.2020.12.006

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


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  9 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.  Dual Purpose Vectors for Rare Neurological Diseases.

Authors:  Brian W Bigger
Journal:  Mol Ther       Date:  2020-09-18       Impact factor: 11.454

Review 3.  Biochemical consequences of mutations causing the GM2 gangliosidoses.

Authors:  D J Mahuran
Journal:  Biochim Biophys Acta       Date:  1999-10-08

4.  Sialidase-mediated depletion of GM2 ganglioside in Tay-Sachs neuroglia cells.

Authors:  S A Igdoura; C Mertineit; J M Trasler; R A Gravel
Journal:  Hum Mol Genet       Date:  1999-06       Impact factor: 6.150

5.  Crystallographic structure of human beta-hexosaminidase A: interpretation of Tay-Sachs mutations and loss of GM2 ganglioside hydrolysis.

Authors:  M Joanne Lemieux; Brian L Mark; Maia M Cherney; Stephen G Withers; Don J Mahuran; Michael N G James
Journal:  J Mol Biol       Date:  2006-04-27       Impact factor: 5.469

6.  Crystal structure of human beta-hexosaminidase B: understanding the molecular basis of Sandhoff and Tay-Sachs disease.

Authors:  Brian L Mark; Don J Mahuran; Maia M Cherney; Dalian Zhao; Spencer Knapp; Michael N G James
Journal:  J Mol Biol       Date:  2003-04-11       Impact factor: 5.469

7.  Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice.

Authors:  Karlaina J L Osmon; Evan Woodley; Patrick Thompson; Katalina Ong; Subha Karumuthil-Melethil; John G Keimel; Brian L Mark; Don Mahuran; Steven J Gray; Jagdeep S Walia
Journal:  Hum Gene Ther       Date:  2016-07       Impact factor: 5.695

8.  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

9.  Construction of a hybrid β-hexosaminidase subunit capable of forming stable homodimers that hydrolyze GM2 ganglioside in vivo.

Authors:  Michael B Tropak; Sayuri Yonekawa; Subha Karumuthil-Melethil; Patrick Thompson; Warren Wakarchuk; Steven J Gray; Jagdeep S Walia; Brian L Mark; Don Mahuran
Journal:  Mol Ther Methods Clin Dev       Date:  2016-03-02       Impact factor: 6.698
  9 in total

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