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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.

 

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

- 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.

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