Abstract
Hereditary blood diseases are disorders due to genetic factors, including hereditary red blood cell disorders, hereditary white blood disorders and hereditary bleeding disorders. These diseases can be diagnosed via gene analysis, but about half of pathogenic gene mutations of these kinds of diseases are still unknown. Currently, replacement therapies are the only effective management for the hereditary blood diseases and gene therapy may be the curative options. Recent years have found encouraging progress in gene therapy, while carcinogenesis and long-term expressions are the major concerns. In this paper, we review the recent progress and unmet needs in hereditary blood diseases.
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