Literature DB >> 28752467

Therapeutics: Gene Therapy for Alpha-1 Antitrypsin Deficiency.

Alisha M Gruntman1, Terence R Flotte2,3,4.   

Abstract

This review seeks to give an overview of alpha-1 antitrypsin deficiency, including the different disease phenotypes that it encompasses. We then describe the different therapeutic endeavors that have been undertaken to address these different phenotypes. Lastly we discuss future potential therapeutics, such as genome editing, and how they may play a role in treating alpha-1 antitrypsin deficiency.

Entities:  

Keywords:  CRISPR/Cas9; Emphysema; Genome editing; Liver disease; SERPINA1

Mesh：

Substances：

Year:  2017        PMID： 28752467     DOI： 10.1007/978-1-4939-7163-3_27

Source DB:  PubMed          Journal:  Methods Mol Biol        ISSN： 1064-3745


  3 in total

Review 1.  Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases.

Authors:  Zeljka Trepotec; Eva Lichtenegger; Christian Plank; Manish K Aneja; Carsten Rudolph
Journal:  Mol Ther       Date:  2018-12-22       Impact factor: 11.454

2.  Is More Better? Promising Biological Effects of Double-Dose Alpha 1-Antitrypsin Therapy.

Authors:  Irina Petrache
Journal:  Am J Respir Crit Care Med       Date:  2019-08-01       Impact factor: 21.405

Review 3.  Alpha-1 antitrypsin deficiency: outstanding questions and future directions.

Authors:  María Torres-Durán; José Luis Lopez-Campos; Miriam Barrecheguren; Marc Miravitlles; Beatriz Martinez-Delgado; Silvia Castillo; Amparo Escribano; Adolfo Baloira; María Mercedes Navarro-Garcia; Daniel Pellicer; Lucía Bañuls; María Magallón; Francisco Casas; Francisco Dasí
Journal:  Orphanet J Rare Dis       Date:  2018-07-11       Impact factor: 4.123
  3 in total

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