Research Strategy
COL6 Fund has a defined scientific course of:
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Utilizing and expanding RNA and DNA based editing technologies to be applied to Wesley's variant and other Collagen VI-related dystrophy mutations.
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Understanding and taking advantage of fibroblast biology to deliver therapeutics.
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As an organization we will also support design of and bring awareness to the need for children sized assistive robotics.
Researching and developing devices, such as exoskeletons. These devices will bring independence and prolonged walking for children with muscle weakness.
We have been working with leading exoskeleton group at Worchester Polytechnic Institute, and will search for other future partnerships. This will help children having all types of neurodegenerative disorders.
Philosophy/Hypothesis
The flow chart below explains the steps needed to establish the science that will support therapy, and eventually produce a cure.
Assistance Research
Academic Development of Light Weight Exoskeleton Device
Product Development
Fibroblast Discovery
Intersitial Fibroblast Collagen VI Activation Research
Allele Specific Genetic
Manipulation
Genetic Models of Collagen VI
Biomarker Discovery
Targeting Strategy
Targetting Strategy for Heterozygous Mutations
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- Antisense
- Genetic editing
Preclinical Modeling
Clinical Trials
Clinical trails will be necessary to bring therapeutics to fruition. We have already been in contact with scientists, industry representatives, and organizations to proceed in all areas of the aforementioned research.