Validation of Clinical-Grade Electroporation Systems for CRISPR-Cas9-Mediated Gene Therapy in Primary Hepatocytes for the Correction of Inherited Metabolic Liver Disease

Hepatocyte transplantation (HTx) combined with ex vivo gene therapy has garnered significant interest due to its potential for treating many inherited metabolic liver diseases. The biggest obstacle for HTx is achieving sufficient engraftment levels to rescue diseased phenotypes, which becomes more c...

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Номзүйн дэлгэрэнгүй

Үндсэн зохиолчид:	Justin Gibson, Abishek Dhungana, Menam Pokhrel, Benjamin Arthur, Pramita Suresh, Olumide Adebayo, Renee N. Cottle
Формат:	Artigo
Хэл сонгох:	Inglês
Хэвлэсэн:	MDPI AG 2025-05-01
Цуврал:	Cells
Нөхцлүүд:	CRISPR-Cas9 clinical translation gene therapy therapeutic liver repopulation electroporation
Онлайн хандалт:	https://www.mdpi.com/2073-4409/14/10/711
Шошгууд:	Шошго нэмэх Шошго байхгүй, Энэхүү баримтыг шошголох эхний хүн болох!