Researchers at USC and Sangamo Biosciences have developed a more efficient genome editing method for HSPCs.
Gene therapy using hematopoietic stem and progenitor cells (HSPCs) has great potential for treating HIV and other diseases of the blood and immune system. One application of gene therapy that is close to clinical translation is the disruption of the HIV-1 co-receptor, CCR5, to engineer cells that are resistant to HIV infection. While promising, the genome editing process isn’t perfect, so only some of the cells end up carrying the desired modification. HSPCs are relatively rare populations that are quiescent, which makes it difficult to transfer genes into these cells with high efficiency. In addition, correction of a mutation or insertion of new DNA sequences at a specific location is often challenging.
