TABLE 2.

Ribonucleoprotein (RNP) mediated delivery of CRISPR/Cas9 in patient derived CD34⁺ HSPCs.

Disease	CRISPR/Cas9 delivery	Efficiency	References
X-Linked Hyper-IgM Syndrome	RNP	∼33%	Kuo et al. (2018)
X-linked chronic granulomatous disease	RNP	>21%	De Ravin et al. (2017)
Fanconi Anemia	RNP	23.33%	Román-Rodríguez et al. (2019)
Wiskott - Aldrich Syndrome	RNP	60%	Rai et al. (2020)
Sickle cell disease	RNP	24.5 ± 7.6%	Park et al. (2019)
Sickle cell disease	RNP	32%	DeWitt et al. (2016)
Severe Congenital Neutropenia	RNP	40–56%	Tran et al. (2020)
β-thalassemia & Sickle cell disease CRISPR/Cas9 clinical trial	RNP	∼80%	Frangoul et al. (2021b)
Mucopolysaccharidosis Type I	RNP	76 ± 8%	Gomez-Ospina et al. (2019)