The pathway to cure
Gene therapy has the potential to provide a definitive solution for those affected by this condition.
What are the steps in our research?
Gene Packaging
The therapeutic gene is packaged into a carrier. Scientists choose AAV9 because it can safely carry the "fixing tool" to the right place in our body.
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2. Preclinical Testing
Rigorous laboratory testing is done to determine the effectiveness and safety of the treatment.
3. Regulatory Review
Before human trials can begin, relevant regulatory bodies (e.g., the FDA in the USA) review the preclinical data. They ensure the potential benefits outweigh the risks.
4. Clinical Trials
If approved, the therapy proceeds to human trials, where it can begin helping people with this disease.
What is gene therapy?
In Mucolipidosis, gene therapy seeks to target the faulty gene responsible for the malfunctioning cell process and replacing or repairing it.
$20/Month
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