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cyclophosphamide + anti-thymocyte globulin + autologous hematopoietic stem cell transplantation
- Development stage
- Unknown
- Lead developer
- Richard Burt
- Modality
- Small Molecules, Antibody-Based Therapeutics, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
A therapeutic procedure involving a non-myeloablative conditioning regimen followed by autologous hematopoietic stem cell transplantation (HSCT), specifically investigated by Dr. Richard Burt for Autoimmune-Related Retinopathy and Optic Neuropathy (ARRON) syndrome. The treatment utilizes high-dose cyclophosphamide and rabbit anti-thymocyte globulin (ATG) to achieve immune ablation, destroying the autoreactive lymphocytes responsible for attacking the retina and optic nerve. Subsequently, the patient's own hematopoietic stem cells are re-infused to reconstitute a new, self-tolerant immune system. This approach aims to halt the progression of vision loss in patients with ARRON syndrome, a rare autoimmune condition characterized by the presence of anti-retinal or anti-optic nerve antibodies.
- Other names
- HSCTImmune Ablation and Hematopoietic Stem Cell Transplantationautologous hematopoietic stem cell transplantationRichard Burt HSCT protocol
02
Targets
DNA
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