Targeting Therapies
Therapy Levels
Flow of Genetic Information
Description
Treatment Targets/
Strategies per Stage
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.
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
c) Testing in Models
Response testing
Tolerability testing
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)
Targeting the removal of mutations causing diseases envisions life long cures.
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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.