Induced Pluripotent Stem Cells and CRISPR-Cas9 Innovations for Treating Alpha-1 Antitrypsin Deficiency and Glycogen Storage Diseases.

Publisher: MDPI Country of Publication: Switzerland NLM ID: 101600052 Publication Model: Electronic Cited Medium: Internet ISSN: 2073-4409 (Electronic) Linking ISSN: 20734409 NLM ISO Abbreviation: Cells Subsets: MEDLINE

Original Publication: Basel, Switzerland : MDPI

alpha 1-Antitrypsin Deficiency*/therapy ; alpha 1-Antitrypsin Deficiency*/genetics ; Induced Pluripotent Stem Cells*/metabolism ; CRISPR-Cas Systems*/genetics ; Glycogen Storage Disease*/genetics ; Glycogen Storage Disease*/therapy ; Glycogen Storage Disease*/metabolism ; Gene Editing*/methods; Humans ; Genetic Therapy/methods ; Animals

Human induced pluripotent stem cell (iPSC) and CRISPR-Cas9 gene-editing technologies have become powerful tools in disease modeling and treatment. By harnessing recent biotechnological advancements, this review aims to equip researchers and clinicians with a comprehensive and updated understanding of the evolving treatment landscape for metabolic and genetic disorders, highlighting how iPSCs provide a unique platform for detailed pathological modeling and pharmacological testing, driving forward precision medicine and drug discovery. Concurrently, CRISPR-Cas9 offers unprecedented precision in gene correction, presenting potential curative therapies that move beyond symptomatic treatment. Therefore, this review examines the transformative role of iPSC technology and CRISPR-Cas9 gene editing in addressing metabolic and genetic disorders such as alpha-1 antitrypsin deficiency (A1AD) and glycogen storage disease (GSD), which significantly impact liver and pulmonary health and pose substantial challenges in clinical management. In addition, this review discusses significant achievements alongside persistent challenges such as technical limitations, ethical concerns, and regulatory hurdles. Future directions, including innovations in gene-editing accuracy and therapeutic delivery systems, are emphasized for next-generation therapies that leverage the full potential of iPSC and CRISPR-Cas9 technologies.

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Keywords: CRISPR-Cas9; alpha-1 antitrypsin deficiency; gene therapy; glycogen storage disease; induced pluripotent stem cells; metabolic disorders; therapeutic modeling

Date Created: 20240626 Date Completed: 20240626 Latest Revision: 20250122

20250122

PMC11201389

10.3390/cells13121052

38920680