Mastodawn

CellBioNews Aug 9, 2024

Study maps how #genes instruct #kidneys to develop differently in #mice and #humans.

https://phys.org/news/2024-08-genes-kidneys-differently-mice-humans.html

Study maps how genes instruct kidneys to develop differently in mice and humans

How similar is kidney development in humans and in the lab mice that form the foundation of basic medical research? In a study published in Developmental Cell, USC Stem Cell scientists probe this question by comparing the activity and regulation of the genes that drive kidney development in lab mice and humans.

Phys.org

Chuck Darwin Jul 12, 2023

Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F

Usher Syndrome Type 1F is an inherited disease that causes profound #hearing #loss from birth and #impairs #vision from adolescence.

At Harvard Medical School and the Ohio State University, researchers have developed a “mini gene” approach that they believe could help to treat the disease. Their study, published in Nature Communications, focuses on protocadherin-15 (#PCDH15), a protein found in the inner ear receptor cells that, when mutated, gives rise to the unraveling of this syndrome.

https://www.nature.com/articles/s41467-023-38038-y

Mini-PCDH15 gene therapy rescues hearing in a mouse model of Usher syndrome type 1F - Nature Communications

Mutations in PCDH15 cause deafness and blindness in Usher syndrome 1 F, but gene therapy is difficult because the PCDH15 sequence is too large for AAV vectors. Here, the authors engineered a miniPCDH15 that fits in AAV and rescues hearing in mouse Usher syndrome 1F models.

Nature