The Official Website of the Acid Maltase Deficiency Association

It is difficult to say what is impossible for the dreams of yesterday are the hopes of today and the reality of tomorrow. - Robert H. Goddard

Gene Therapy: National Institutes of Health

Posted on: June 01, 1998

National Institutes of Health
Paul Plotz, M.D., Nina Raben, M.D., Ph.D.
Nina Raben, M.D., Ph.D., NIH Research Chemist, visited the Netherlands to collaborate with Dr. Arnold Reuser on genetic mutations in AMD. They studied the presence of certain disease related mutations, how they affect the mechanisms of the acid maltase enzyme, and how transfers are made within the cells. The NIH has developed a mouse model for Acid Maltase Deficiency. (April, 1997; updated June. 1998)

The NIH in conjunction with Tel Aviv University, Israel, published an abstract on gene therapy relating to Type II Glycogen Storage Disease or Acid Maltase Deficiency. It was published in:
Human Gene Therapy 8:1555-1563 (September 1, 1997).

“Retroviral Transfer of Acid a-Glucosidase cDNA to Enzyme-Deficient Myoblasts Results in Phenotypic Spread of the Genotypic Correction by Both Secretion and Fusion”

See abstract-Zaretsky (September 1, 1997)

Disclaimer: The AMDA does not endorse any of the products, medications, treatments or information reported herein. The website and its contents is intended for informational purposes, only. We strongly advise that you discuss all medications, treatments, and/or products with your physician.

Address: AMDA PO Box 700248
San Antonio, Texas 78270 USA