Table 1.

Genetic diseases of blood cells and the transplantation modalities that have been applied clinically as therapies or are in pre-clinical development.

Category of Disease	Specific Conditions	Transplantation Modalities Applied
		Allogeneic HSCT	γ-Retroviral Gene Therapy	Lentiviral Gene Therapy	Genome Editing
Primary Immune Deficiencies	ADA-Deficient Severe Combined Immune Deficiency	+	+	+
	X-linked Severe Combined Immune Deficiency	+	+	+	Pre-clinical
	Other Genetic Forms of SCID (Artemis, Rag1/2).	+		Pre-clinical
	Wiskott-Aldrich Syndrome	+	+	+	Pre-clinical
	Chronic Granulomatous Disease	+	+	+	Pre-clinical
	Leukocyte Adhesion Deficiency	+	+	Pre-clinical
	Hemophagocytic Lymphohistiocytosis	+		Pre-clinical
	X-linked Hyper IgM Syndrome	+			Pre-clinical
	X-linked Lymphoproliferative Disease	+
	X-linked Agammaglobulinemia	Few			Pre-clinical
	Common Variable Immunodeficiency	Heterogeneous Genetic Etiologies, Often Unknown. May Require Editing
Hemoglobinopathies	Sickle Cell Disease	+		+	Pre-clinical
	β-thalassemia	+		+	Pre-clinical
Storage and Metabolic Disorders	Gaucher Disease and other lipidoses	+	+
	Mucopolysaccharidoses (I-VII)	+		+
	X-linked Adrenoleukodystrophy	+		+ Recent Phase II/III
	Metachromatic Leukodystrophy	+		+
	Osteopetrosis	+		Pre-clinical
Congenital Cytopenias and Stem Cell Defects	Fanconi’s Anemia	+	+	+	Pre-clinical
	Schwachman-Diamond Syndrome	+		Pre-clinical	Pre-clinical
	Kostmann’s Syndrome	+