Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells
Launched by NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE (NINDS) · Nov 3, 1999
Trial Information
Current as of March 27, 2025
Completed
Keywords
ClinConnect Summary
This protocol was developed in order to obtain bone marrow stem cells for ex vivo transduction with retroviruses containing the human glucocerebrosidase gene. We continue to enter a small number of patients to this protocol each year. Studies with the bone marrow hematopoietic progenitor cells have enabled us to identify the most effective retroviral construct currently available in order to carry out gene therapy trials in patients with Gaucher's disease. The data revealed that a comparatively simple retroviral construct containing human glucocerebrosidase cDNA driven by the MoLV promoter ...
Gender
ALL
Eligibility criteria
- • Normal and patient volunteers.
- • Individuals with platelet counts less than 40,000/ul, PT greater than 15 seconds, or PTT greater than 40 seconds will not undergo bone marrow aspiration.
- • Individuals with hematologic disorders other than Gaucher Disease, Fabry Disease, or mild iron deficiency will not undergo bone marrow aspiration.
- • HIV positive individuals will be excluded from participating.
About National Institute Of Neurological Disorders And Stroke (Ninds)
The National Institute of Neurological Disorders and Stroke (NINDS) is a leading component of the National Institutes of Health (NIH), dedicated to advancing our understanding of the brain and nervous system through innovative research. NINDS sponsors and conducts clinical trials aimed at developing effective treatments and improving patient outcomes for a wide range of neurological disorders, including stroke, epilepsy, multiple sclerosis, and neurodegenerative diseases. By fostering collaboration among researchers, healthcare providers, and patient communities, NINDS plays a pivotal role in transforming scientific discoveries into clinical applications, ultimately enhancing the quality of life for individuals affected by neurological conditions.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Bethesda, Maryland, United States
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
Discussion 0
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