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JME Group
Oral enzyme replacement therapy (Oral-ERT) and
non-viral gene replacement therapy for human and animal conditions.
Treatments
1. To investigate the feasibility and commercialization of a novel approach for enzyme replacement (ERT) via oral administration (Oral-ERT) of a recombinant human acid maltase (rhGAA) produced in transgenic tobacco seed in GAA knockout mice
2. To study valproic acid (VPA) to rescue the IVS1 splice mutation in different cells lines including skeletal muscle and the GAA KO mouse containing this mutation thus providing in vitro and in vivo proof of principle for the use of VPA to rescue splicing of c.-32-13 t>g allele. VPA may be a short-term cure for patients with this mutation directly or in combination with Myozyme or other rGAAs our company is developing besides dramatically reducing costs of treatment.
3. To investigate the feasibility and commercialization of a novel approach for ERT via oral administration of ground germinated barley (GBB) in GAA KO mice. Eventual use in POMPE patients will be to maintain a sustained, therapeutic level of enzyme on a daily basis to improve the efficacy of treatment for the disease
4. To investigate intranasal non-viral gene delivery with different carriers including naked plasmid or plasmid carried by crotamine-a CPP (cell penetrating peptide), liposome and PEI25/Pluronic F-68 into cells from patients and to the brain in GAA KO mice. This therapy will allow the delivery of the normal acid maltase to the brain and will potential supplement enzyme replacement therapy.
5. To develop other treatments besides ERT for POMPE disease , other types of therapy (gene therapy, diet, exercise, etc.) must be explored and evaluated. Crotamine non-viral gene delivery besides transducing tissues, may result in the recombinant protein secreted into the circulation that can be taken up by distant tissues and cells. Thus, it may serve as a combination of ERT via gene delivery or as "factories" to produce the recombinant protein
6. Food or dietary supplement to improve activity and health for-
- athletes, exercise and elderly.
7. Treatment of Fabry’s disease with oral administration (Oral-ERT) of a recombinant human α-galactosidase A (tobrhGALA) produced in transgenic tobacco seeds.
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