Rare Disorder Treatment via Gene therapy and Stem cell therapeutics.
Human genome consist of 6 billion nucleotides a single mutation in nucleotide causes life threatening rare disease like Lesch-Nyhan syndrome , Adenosine deaminase deficiency related severe combined immunodeficiency (ADA-S), Gaucher disease type III (GD), X-linked chronic granulomatous disease (X-CGD), A1-antitrypsin deficiency. These monogenic diseases require constant and expensive care but by therapeutics techniques in gene editing by insertion, deletion or modification in these mutated nucleotide sequences can cure these disease from root level This can be possible only by gene therapy. These can be induced by replacing, inactivating and changing the sequence of the mutated gene. Hence gene therapy and genome editing is the only alternative approach to cure form rare diseases.
· Lesch-Nyhan syndrome
· Gaucher disease type III (GD)
· X-linked chronic granulomatous disease (X-CGD)
· A1-antitrypsin deficiency
· Adenosine deaminase deficiency related severe combined immunodeficiency