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. Author manuscript; available in PMC: 2019 Jul 3.
Published in final edited form as: Methods Mol Biol. 2015;1239:39–73. doi: 10.1007/978-1-4939-1862-1_4

Table 1.

Recombinagenic and mutagenic frequencies of therapeutic targeting and editing of genomic loci to ameliorate disease etiologies

Disease etiology Genomic locus Recombinagenic and mutagenic molecules Cell type Molecular delivery modality Recombinagenic and mutagenic frequency (%)
Beta-thalassemia Beta-globin Donor DNA CHO electroporation 0.05
Beta-thalassemia Beta-globin Donor DNA + triplex-forming PNA CHO electroporation 0.20
Hemoglobinopathies, such as sickle cell disease Gamma-globin Donor DNA K562 electroporation 0.29
Hemoglobinopathies, such as sickle cell disease Gamma-globin Donor DNA + triplex-forming PNA K562 electroporation 1.63
HIV-1 CCR5 Donor DNA + triplex-forming PNA hCD34+ nucleofection 0.10
HIV-1 CCR5 Donor DNA + triplex-forming PNA hCD34+ nanoparticles 0.91
HIV-1 CCR5 Donor DNA + triplex-forming PNA THP-1 electroporation 2.46

Recombinagenic and mutagenic frequencies on the chromosome or episome are influenced by a number of factors, including the particular endogenous genomic locus, exogenously introduced molecules, cell type, and molecular delivery modality