Choosing Targets for Gene Therapy

摘要: Gene therapy is often attempted in fatal diseases with no known cure, or after standard therapies have failed. Targeting gene defects includes addressing a single mutation, multiple mutations several genes, even missing extra copies particular disease. A defect one specific may impair normal function of the corresponding expressed protein. For example, X-linked severe combined immunodeficiency (X-SCID), there mutation IL2 receptor ┛ gene. Another classic example occurs thalassemia propagated by ┚-globulin Some are caused genes. some cardiovascular manifest due to different chromosomes which result inherited environmental factors. Before approaching disease using therapy, key protein(s) and pathways involved should first be identified. However, cases an abnormal formed that results disease; such case for BcrAbl The oncogenic Bcr-Abl protein causative agent chronic myelogenous leukemia (CML) could blocked CML treatment. Genomic sequencing information, microarrays, biochemical assays can used determine upor downregulated proteins disease, will help these proteins. In cancers, signal transduction oncogenesis been mapped out, allowing hub Hub essential interact other signaling cascades. If selected properly, adding back tumor-suppressing (such as p53), blocking survivin) halt cancer alter progression. mislocalization cause cancer; this exploited approaches. Further, new types being developed our lab direct cellular compartments where their altered. This chapter summarize targets also focus on choosing newer therapy.