| A | Adenine |
| AAV6 | Adeno-associated virus 6 |
| ABEs | Adenine base editors |
| ANZCTR | Australia and New Zealand Clinical Trials Registry |
| BCL11A | B-cell lymphoma/leukemia 11A |
| BEs | Base editors |
| BT | β-thalassemia |
| C | Cytosine |
| Cas | Clustered regularly interspaced short palindromic repeats (CRISPR)-associated |
| CBEs | Cytosine base editors |
| ChiCTR | Chinese Clinical Trial Registry |
| CMN | CRISPR Medicine News |
| CRISPR | Clustered regularly interspaced short palindromic repeats |
| DSB | Double-stranded break |
| G | Guanine |
| gRNA | guide RNA |
| HCT | Hematopoietic cell transplant |
| HDR | Homology-directed repair |
| HPFH | Hereditary persistence of fetal hemoglobin |
| HSC | Hematopoietic stem cell |
| HSPC | Hematopoietic stem and progenitor cells |
| IHBDS | Institute of Hematology and Blood Diseases |
| indels | Insertions or deletions |
| LGMD | Limb–girdle muscular dystrophy |
| nCas9 | Cas9 nickase |
| NHEJ | Non-homologous end joining |
| NIAID | National Institute of Allergy and Infectious Diseases |
| NIH | National Institutes of Health |
| PAM | Protospacer adjacent motif |
| pegRNA | prime-editing guide RNA |
| PKD | Pyruvate kinase deficiency |
| RT | Reverse transcriptase |
| SCD | Sickle cell disease |
| SCID | Severe combined immunodeficiency |
| T | Thymine |
| TDT | Transfusion-dependent β-thalassemia |
| X-CGD | X-linked chronic granulomatous disease |
| X-SCID | X-linked severe combined immunodeficiency |
| ZFN | Zinc-finger nuclease |