Rare genetic diseases are conditions caused by mutations in a person’s DNA that occur in fewer than 1 in 2,000 people. Symptoms vary widely depending on the disease, but they often involve developmental, metabolic, or neurological dysfunctions. Worldwide, about 7,000 rare diseases affect approximately 400 million people. The market for rare disease treatments is valued at $200 billion and is expected to expand as more therapies are developed. Many of these diseases have no approved treatments. For companies, developing drugs for rare genetic diseases is a high-reward venture due to orphan drug incentives, high unmet medical need, and less competition compared to more common diseases.
Zebrafish Model: Generating knockdown zebrafish by using gene specific morpholino (antisense oligonucleotide) at 1 to 4 cell stage embryos and characterizing the induced phenotype to identify the compounds which show rescue effect and explore novel therapeutic strategies