In 1997 the Niemann-Pick type C
gene
was cloned
at the National Institute of Heath. The
research is now aimed at determining the function of the
protein that the mutated
gene
codes for. Once this is done, scientist can then test drugs that may
accomplish the job that the non-functional
protein should be doing, and thereby develop a cure.
The
gene
that is altered in the majority of patients with type C (and D) was identified by investigators at NINDS (National Institute of Neurological Disorders and Stroke). A second (different)
gene
that is mutated in a minority of patients with type C Niemann-Pick disease was discovered in 2000.
Also, once the NP gene is well studied,
gene therapy will be
developed and will serve as a cure for all the
different types of NP mutations that exist.
All of these
research programs are very expensive, and can not continue without your help. Please click on one of the links below to make a donation to one of the foundations. |
