«This finding really excites us because it makes us think that this is
a gene repair system that could be used to treat a range of diseases — not just tyrosinemia but others as well,» says Daniel Anderson, associate professor in MIT's Department of Chemical Engineering and a member of MIT's Koch Institute for Integrative Cancer Research and Institute for Medical Engineering and Science (IMES).
Not exact matches
Researchers engineered a virus to carry a replacement
gene to
repair the immune
systems of the sick children.
They programmed the new CRISPR / Cas9
system to snip out the dysfunctional exon, leaving the body's natural
repair system to stitch the remaining
gene back together to create a shortened — but functional — version of the
gene.
In the current work, they used a new variation of the
gene - editing
system to
repair the defect in both a mouse model and in human cells.
In the latest experiments the Mitalipov group focused on snipping out the mutated
gene in heterozygous cells — a situation in which there was still a «good» nonmutated copy available for the natural cellular
repair systems in the embryo to use as a template for
repair after the researchers edited out the problematic one.
With her HHMI money, she will explore the connections between the immune
system and circadian clock
genes, as well as the link between immune response and DNA
repair genes.
Field reports suggest that not all K13 mutations are capable of causing resistance, and the genetic
system developed by Dr. Fidock to study K13, based on DNA
repair approaches that are being used in human
gene therapy studies, will be critical in identifying real hot spots of resistance.
But, fascinating anecdotes aside, does hard evidence exist of specific vulnerability
genes or how they might impair the immune
system, brain, and gut — and most important, do we have any rational, reliable approaches to help
repair the damage?
The mfd
gene was cloned and sequenced and the Mfd protein was purified and used to reconstitute strand - specific
repair in a completely defined
system.
However, at least 16 % or more can not be analyzed with these methods because individuals have a mutation in another mismatch
repair gene, PMS2 (PMS 1 homolog 2, mismatch
repair system component).
Contact: 508-685-3525;
[email protected] Woods Hole, Mass. — Many of the
genes involved in natural
repair of the injured spinal cord of the lamprey are also active in the
repair of the peripheral nervous
system in mammals, according to a study by a collaborative group of scientists at the Marine Biological Laboratory (MBL) and other institutions.
PER2: Deletion of the PER2
gene in mice, associated with the mechanisms of circadian rhythm, appears to improve DNA
repair in stem cell populations relevant to the immune
system, resulting in a healhier immune cell population, better immune function in old age, and a modestly extended life span.
Over time, the
repair system in the cell works worse, and much of this damage either is not corrected at all, disrupting the expression of
genes, or converted into mutations.
The Yale team found more than 580 different
genes that may play a role in regeneration of axons in nerve cells, something that rarely occurs in adult mammals but is of vital interest to scientists hoping to
repair injuries to the central nervous
system.