Progress in the molecular biology of inherited bleeding disorders

摘要

Since the advent of recombinant DNA technology in the early 1980s, the application of molecular biological approaches to haemophilia treatment has shown progressive advancement. More recently, the addition of various forms of cellular therapies to complement gene and protein-based therapeutics has further enhanced the potential for innovative treatments of these conditions.In this chapter, we have summarized the recent trends pertaining to several aspects of novel protein, gene, and cell-based therapeutic strategies for haemophilia.