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Encapsulation cell therapy for mucopolysaccharidosis type VII using genetically engineered immortalized human
Hideyuki Nakama1, Keiko Ohsugi, Taisuke Otsuki
1Department of Neurosurgery, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashicho, Kodaira, Tokyo 187-8551, Japan. nakama@ncnp.go.jp
The Tohoku Journal of Experimental Medicine
|April 26, 2006
Summary
Mucopolysaccharidosis type VII (MPSVII) is a rare genetic disorder. Encapsulated, engineered human amniotic epithelial cells effectively delivered beta-glucuronidase, showing promise for treating MPSVII, particularly in the brain.
Area of Science:
- Biochemistry
- Genetics
- Cell Biology
Background:
- Mucopolysaccharidosis type VII (MPSVII) is a lysosomal storage disease caused by beta-glucuronidase (GUSB) deficiency.
- GAG accumulation in MPSVII leads to lysosomal distension, impacting multiple tissues, including the central nervous system (CNS).
- Current therapies struggle to cross the blood-brain barrier (BBB) or carry risks like tumor formation.
Purpose of the Study:
- To develop a novel cell-based therapy for MPSVII using encapsulated, genetically engineered cells.
- To overcome the limitations of existing treatments in delivering GUSB to the CNS.
Main Methods:
- Generated immortalized human amniotic epithelial (IHAE) cells engineered to over-express and secrete human GUSB.
- Encapsulated these IHAE cells to prevent immune rejection and control GUSB expression.
- Evaluated therapeutic efficacy in vitro and in vivo using C3H mice.
Main Results:
- Encapsulated IHAE cells secreted significant amounts of GUSB in vitro.
- GUSB was detected in the cerebral parenchyma of treated mice seven days post-implantation.
- Demonstrated successful delivery of GUSB across the BBB via encapsulated cells.
Conclusions:
- Encapsulation cell therapy with genetically engineered IHAE cells is a viable strategy for MPSVII treatment.
- This approach effectively delivers GUSB to the brain, addressing a key challenge in MPSVII therapy.
- Offers a promising new armamentarium for managing MPSVII, particularly CNS manifestations.
