New Gene Editing Findings from State University of New York (SUNY) Binghamton Published (Induced Pluripotent Stem Cells and CRISPR-Cas9 Innovations for Treating Alpha-1 Antitrypsin Deficiency and Glycogen Storage Diseases).

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      BINGHAMTON (N.Y.)
    • Abstract:
      A recent article from Stem Cell Week discusses the use of induced pluripotent stem cells (iPSCs) and CRISPR-Cas9 gene editing technology in the treatment of metabolic and genetic disorders. The researchers highlight how iPSCs can be used for disease modeling and pharmacological testing, while CRISPR-Cas9 offers precise gene correction for potential curative therapies. The article specifically focuses on the treatment of alpha-1 antitrypsin deficiency (A1AD) and glycogen storage disease (GSD), which impact liver and pulmonary health. The researchers also address challenges such as technical limitations, ethical concerns, and regulatory hurdles. They emphasize the need for future innovations in gene-editing accuracy and therapeutic delivery systems. [Extracted from the article]
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