The Jeremiah Metzger Lecture: Turning Genes Into Medicines: Highlights and Hurdles in the Development of Gene Therapy for Genetic Disease.
Year: 2023
Type: article
Abstract: deficiency, a key to success was development and validation of a novel clinical endpoint for a disease that previously lacked a pharmacologic treatment.
Source: PubMed Central
Author: Katherine A High
Language: English
Subfield: Genetics
Domain: Life Sciences
Open Access status: green
OpenAlex ID: W4386705487