Two French babies who were born with a rare immune deficiency have apparently been
cured by gene therapy.
Since then, he has begun giving university lectures about the importance of exercising caution as a clinical scientist, especially when it comes to stem cells, which today have the cachet once
held by gene therapy.
That enzyme turned out to be defective in the short - lived strain of roundworms, but it worked fine in normal worms and in strains
rescued by gene therapy.
Based on these promising results, work has begun on the necessary developments to propose to patients suffering from Friedreich's ataxia and presenting a progressive cardiomyopathy a
treatment by gene therapy.
«Our next step is to take the gene which codes for tyrosinase, and put this into liposomes to see if we can influence hair
colour by gene therapy,» says Hoffman.
Mice in which telomeres have been
lengthened by gene therapy tended to live longer in some studies than mice that didn't get the treatment.
In previous work, Dr. Ryosei Minoda, head of the Department of Otolaryngology at Kumamoto General Hospital, reported that hearing loss was
restored by gene therapy to the inner ear of Connexin 30 deficient fetal mice.
Whereas Humira alone could have caused the immune suppression, another possibility is that Humira combined with the TNF - α blocker
encoded by the gene therapy pushed Mohr over the edge.
THE PROSPECT of curing some
diseases by gene therapy is coming closer, with the publication this month of three reports of possible ways of treating cancer, immune deficiency and Duchenne muscular dystrophy.
Yet, their efforts to rescue cone
function by gene therapy in mid-course LCA stages failed: when applied to 35 - day old Rpe65 - deficient mice, gene therapy restores rod function only (Pang et al. 2010).
ORDINARY cells from people with a genetic disease can be «fixed»
by gene therapy and then reprogrammed to be stem cells that will produce a limitless supply of defect - free cells.