Engineered IRES-mediated promoter-free insulin-producing cells reverse hyperglycemia.

ABSTRACT:

Background

Endogenous insulin supplementation is essential for individuals with type 1 diabetes (T1D). However, current treatments, including pancreas transplantation, insulin injections, and oral medications, have significant limitations. The development of engineered cells that can secrete endogenous insulin offers a promising new therapeutic strategy for type 1 diabetes (T1D). This approach could potentially circumvent autoimmune responses associated with the transplantation of differentiated β-cells or systemic delivery of viral vectors.

Methods

We utilized CRISPR/Cas9 gene editing coupled with homology-directed repair (HDR) to precisely integrate a promoter-free EMCVIRES-insulin cassette into the 3' untranslated region (UTR) of the GAPDH gene in human HEK-293T cells. Su

SUBMITTER: Li Y 

PROVIDER: S-EPMC11392723 | biostudies-literature | 2024

REPOSITORIES: biostudies-literature