CRISPR/Cas9 Genome Editing to Treat Sickle Cell Disease and B-Thalassemia: Re-Creating Genetic Variants to Upregulate Fetal Hemoglobin Appear Well-Tolerated, Effective and Durable.

Autor: Lin, Michelle I., Paik, Elizabeth *, Mishra, Bibhu *, Burkhardt, David *, Kernytsky, Andrew *, Pettiglio, Michael *, Chen, Yi-Shan *, Tomkinson, Kaleigh *, Woo, Amanda *, Cortes, Mauricio *, Tan, Siyuan *, Borland, Todd D *, Klein, Lawrence *, Yen, Angela *, Mahajan, Sudipta *, Chan, Eric *, Eustace, Brenda *, Porteus, Matthew, Chakraborty, Tirtha *, Cowan, Chad *, Novak, Rodger *, Lundberg, Ante
Zdroj: In Blood 8 December 2017 130 Supplement 1:284-284
Databáze: ScienceDirect