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Targeting Therapies

Therapy Levels 

Flow of Genetic Information

Description

Treatment Targets/
Strategies per Stage

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The foundational level of our genetic information. Permanent changes to the person.

Carries instructions from DNA

to the cell. Constantly
produced and recycled.

Workhorses/machinery of the
cell responsible for nearly
every task of life.

The human body is composed
of trillions of cells that can group together to make organs.

CRISPR/Prime Editing,

Gene Therapy

Anti-sense Oligonucleotide

(Splice Modulating and Knockdown),

U7 snRNA

Targeted Protein Degradation (PROTAC),

Collagen VI Release Strategies

Interstitial Fibroblast Therapies,

Muscle Contractures Treatments

Medicine,

Robotic Assistance

RNA Therapy (Antisense Oligonucletides ASO) Development

RNA targeting therapies are already being used to successfully treat neuromuscular diseases.

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a)  DNA Analysis and RNA Therapy Design

Does the therapy only target altered Collagen VI RNA?

Will the therapy withstand environment in the human body?

b) Production of RNA Therapy

Testing of response and dosing

Safety testing

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c) Testing in Models

Response testing

Tolerability testing

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At completion we will contact

FDA for movement/requirements to a start a Clinical Trial

DNA Editing Therapy 

A new class of potential DNA editing therapies are entering the scene for precision medicine

(CRISPR, Prime Editing, Base Editing, and more)

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Targeting the removal of mutations causing diseases envisions life long cures.

We will invest in research to see if the editing at the DNA level of one mutation in Collagen VI can reverse the disease. In the laboratory and mouse models. As this exciting therapeutic option enters humans we hope to be close to the forefront of any applications in Collagen VI patients.

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