Gene therapy fully restores vision in mouse model of Leber congenital amaurosis
Mice lacking the protein retGC1, which is deficient in humans suffering Leber congenital amaurosis-1 (LCA1), a disorder that causes severe visual impairment beginning in infancy, received gene therapy to replace retGC1 and showed fully restored visual function that persisted for at least 6 months. The success of this approach strongly support clinical testing of a gene therapy targeted to the retinas of LCA1 patients, conclude the authors of the study published in Human Gene Therapy. The article is available free on the Human Gene Therapy website until September 30, 2015.
Stem Cell Research
-
In some genetic cases of microcephaly, stem cells fail to launch
-
Embryonic gene Nanog reverses aging in adult stem cells
-
Gene controls regeneration of injured muscle by adult stem cells
-
Breakthrough in scaling up life-changing stem cell production
-
New procedure allows long-term culturing of adult stem cells
-
Stem cell transplant from young to old can heal stomach ulcers
-
Scientists discover oldest plant root stem cells
-
Penn bioengineers show why lab-made stem cells might fail: Errors in DNA folding
-
Stem cell therapy improves outcomes in severe heart failure
-
A quartet of genes controls growth of blood stem cells
-
New way to harvest stem cells better for donors
-
Oncogene controls stem cells in early embryonic development
-
Hacking the programs of cancer stem cells
-
Combining adult stem cells with hormone may speed bone fracture healing
-
Discovery of an embryonic switch for cancer stem cell generation