Ataxia-Telangiectasia Gene
Filed on 1995-05-16
For the development of gene therapy as a clinically useful procedure for training genetic diseases.
Awardee
National Human Genome Research Institute
Currently, there are no FDA-approved therapies for Niemann-Pick disease type-C1 (NPC). NPC is a rare lethal genetic lysosomal storage disorder that results in an accumulation of cholesterol in the liver and spleen and eventually leads to neurodegeneration. 2-hydroxypropyl-β-cyclodextrin (HPβCD) is a cyclodextrin typically used by the pharmaceutical industry as an excipient. Studies of NPC in animal models have shown that HPβCD can reduce the biochemical burden associated with NPC, improving neurological pathology, decreasing neurological dysfunction, and increasing lifespan.
Dr. Campbell began his career with the U.S. Patent and Trademark Office, where he first worked as a patent examiner for four years and was subsequently promoted to a primary patent examiner, a position in which he served for five years. During his tenure there, Dr. Campbell allowed over 400 patents, specializing in a variety of physiological conditions used for nucleic acid detection via hybridization, nucleic acid amplification (PCR), detection of tumors/cancer via mutations, recombinant polypeptides, human genes, and bioinformatics. Dr.