Integrated AAV optimization enables efficient gene delivery to kidney in murine and human tissue

Abstract excerpt

<title>Abstract</title> <p> Unbiased screening of patients seen in nephrology clinics indicate that ~ 10% of patients have a monogenetic cause of kidney disease. For these patients and possibly for the broader diaspora of patients living with chronic kidney disease, gene therapy may be efficacious and possibly curative. A major limitation of gene therapy to the kidney parenchyma thus far, has been seemingly poor...

Topics

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• Animal Virus Infections Studies
• CAR-T cell therapy research
• CRISPR and Genetic Engineering
• Renal and related cancers
• Viral gastroenteritis research and epidemiology
• Viral Infectious Diseases and Gene Expression in Insects
• Virus-based gene therapy research
• Xenotransplantation and immune response

Identifiers and source

Literature Corpus work

bd06f4c5-c9bc-5ee0-af15-c18717aa004a

DOI

10.21203/rs.3.rs-7925411/v1