Investigational Gene-Based Therapy for Alpha-1 Antitrypsin (AAT) Deficiency Demonstrates Durable Response at Five Years

Research led by scientists from the University of Massachusetts Medical School presented at the American Society of Gene and Cell Therapy (ASGTC) Annual Meeting WASHINGTON, May 05, 2016 -- (Healthcare Sales &Marketing Network) -- Researchers from the U... BiopharmaceuticalsApplied Genetic Technologies, gene therapy, Alpha-1 Antitrypsin
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news

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Human Gene Therapy,Volume 30, Issue 8, Page 985-998, August 2019.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
TH and MW contributed equally to this work.BackgroundMultiple myeloma is caused by an accumulation of malignant plasma cells in the bone marrow. Myeloma is characterized by an osteolytic bone disease, caused by increased bone degradation and reduced bone formation. Bone morphogenetic proteins (BMPs) are members of the transforming growth factor (TGF)-β superfamily. BMP-signaling is important for both pre- and postnatal bone formation. Additionally, several BMPs induce growth arrest and apoptosis in myeloma cells. Thus, increasing BMP-signaling in myeloma patients may reduce tumor growth and restore bone formation. We ...
Source: Blood - Category: Hematology Authors: Tags: 652. Myeloma: Pathophysiology and Pre-Clinical Studies, excluding Therapy: Poster I Source Type: research
Human Gene Therapy,Volume 29, Issue 8, Page 843-844, August 2018.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Human Gene Therapy, Ahead of Print.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9, Published online: 27 March 2018; doi:10.1038/s41434-018-0003-1Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9
Source: Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Nuclease-Mediated Gene Therapies for Inherited Metabolic Diseases of the Liver

. Yale J Biol Med. 2017 Dec;90(4):553-566 Authors: Bryson TE, Anglin CM, Bridges PH, Cottle RN Abstract Inherited metabolic diseases (IMDs) of the liver represent a vast and diverse group of rare genetic diseases characterized by the loss or dysfunction of enzymes or proteins essential for metabolic pathways in the liver. Conventional gene therapy involving adeno-associated virus (AAV) serotype 8 vectors provide therapeutically high levels of hepatic transgene expression facilitating the correction of the disease phenot...
Source: The Yale Journal of Biology and Medicine - Category: Universities & Medical Training Tags: Yale J Biol Med Source Type: research
In this report we describe the appearance of...
Source: Proceedings of the National Academy of Sciences - Category: Science Authors: Tags: Biological Sciences Source Type: research
Human Gene Therapy , Vol. 0, No. 0.
Source: Human Gene Therapy - Category: Genetics & Stem Cells Authors: Source Type: research
Annals of the American Thoracic Society,Volume 13, Issue Supplement_4, Page S352-S369, August 2016.
Source: Annals of the American Thoracic Society - Category: Respiratory Medicine Authors: Source Type: research
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