Evaluation of diversity indices to estimate clonal dominance in gene therapy studies.

ABSTRACT: In cell and gene therapy, achieving the stable engraftment of an abundant and highly polyclonal population of gene-corrected cells is one of the key factors to ensure the successful and safe treatment of patients. Because integrative vectors have been associated with possible risks of insertional mutagenesis leading to clonal dominance, monitoring the relative abundance of individual vector insertion sites in patients' blood cells has become an important safety assessment, particularly in hematopoietic stem cell-based therapies. Clinical studies often express clonal diversity using various metrics. One of the most commonly used is the Shannon index of entropy. However, this index aggregates two distinct aspects of diversity, the number of unique species and their relative abundance. This p

SUBMITTER: Corre G 

PROVIDER: S-EPMC10220254 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature