Table 1.
MONOALLELIC - HETEROZYGOUS | BIALLELIC - HOMOZYGOUS | ||||
---|---|---|---|---|---|
SNCA (PARK1) | A53T | Autosomal dominant | Prime editing | 3 | - |
A30P | Autosomal dominant | Prime editing | 3 | 1 | |
PRKN (PARK2) | EX3DEL | Autosomal recessive | CRISPR/Cas9 (dual guide) | - | 3 |
PINK1 (PARK6) | Q129X | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 3 |
DJ1 (PARK7) | EX1–5DEL | Autosomal recessive | CRISPR/Cas9 (dual guide) | - | 3 |
LRRK2 (PARK8) | G2019S | Autosomal dominant | Prime editing, TALEN and CRISPR/Cas9 (HDR) | 4 | 1 |
ATP13A2 (PARK9) | FS | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 5 |
FBXO7 (PARK15) | R498X/FS | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 1 |
DNAJC6 (PARK19) | c.801–2 A>G/FS | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 2 |
SYNJ1 (PARK20) | R258Q/FS | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 2 |
VPS13c (PARK23) | W395C/FS | Autosomal recessive | CRISPR/Cas9 (HDR) | - | 4 |
A444P/FS | Autosomal recessive | CRISPR/Cas9 (HDR) | 1 | 2 | |
GBA1 | IVS2+1/FS | Autosomal dominant1 | CRISPR/Cas9 (HDR) | 4 | 2 |
Wild type | - | - | Parental + 3 independent clones | ||
Edited wild type (EWT) | - | - | Prime editing | Prime editing (6) + CRISPR/Cas9 (2) |
mixed inheritance.