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Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine
Published on: November 4, 2018
Pediatric Liver Diseases: Next-Generation Therapies
Jeff Teckman1, Ashlesha Bagwe2
1Division of Pediatric Gastroenterology and Hepatology, Saint Louis University School of Medicine, Cardinal Glennon Children's Hospital, 1325 South Grand Boulevard, 7th Floor, Saint Louis, MO 63104, USA.
Abstract:
With recent advances in molecular medicine, there has been a transformation of the landscape of pediatric liver disease therapeutics. The advent of gene editing technologies, RNA therapeutics, and molecular chaperone therapies has led to precise targeting of liver pathology. This review aims to shed light on recent breakthroughs in liver-directed nucleic acid therapies such as small interfering RNA, mRNA editing, and CRISPR-based approaches, with a special focus on their application in alpha-1 antitrypsin deficiency, hepatitis B, cystic fibrosis-related liver disease, metabolic dysfunction-associated steatotic liver disease, Alagille syndrome, progressive familial intrahepatic cholestasis, and acute hepatic porphyria.
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