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. 2026 Apr 9;27(8):3384. doi: 10.3390/ijms27083384
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