Creation of de novo cryptic splicing for ALS and FTD precision medicine.
Wilkins OG, Chien MZYJ, Wlaschin JJ, Barattucci S, Harley P, Mattedi F, Mehta PR, Pisliakova M, Ryadnov E, Keuss MJ, Thompson D, Digby H, Knez L, Simkin RL, Diaz JA, Zanovello M, Brown AL, Darbey A, Karda R, Fisher EMC, Cunningham TJ, Le Pichon CE, Ule J, Fratta P
Science. 2024 Oct 4;386(6717):61-69. doi: 10.1126/science.adk2539. Epub 2024 Oct 3.
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Plasmids from Article
| ID | Plasmid | Purpose |
|---|---|---|
| 216152 | TDP-REGv2(mScarlet-FLAG)#7 in pTwist-CMV backbone | Expresses mScarlet in cells with TDP-43 loss-of-function. This plasmid has one of the best dynamic ranges of those tested (~100-fold) in SK-N-BE2 cells. It uses a cryptic exon. [Code 'A11'] Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216153 | TDP-REGv2(mScarlet-FLAG)#9 in pTwist-CMV backbone | Expresses mScarlet in cells with TDP-43 loss-of-function. Has one of the best dynamic ranges of those tested (~100-fold) in SK-N-BE2 cells (brighter than #7). It uses a single intron. [Code 'B11']. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216154 | TDP-REGv2(mScarlet-FLAG)#10 in pTwist-CMV backbone | Expresses mScarlet in cells with TDP-43 loss-of-function. Leaky but very sensitive to mild TDP-43 loss of function. It uses a cryptic exon. [Code 'B5'] Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216155 | TDP-REGv2(mScarlet-FLAG)#11 in pTwist-CMV backbone | Expresses mScarlet in cells with TDP-43 loss-of-function. Very bright and decent dynamic range (>10-fold), slightly leaky. It uses a single intron. [Code 'B12']. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216156 | TDP-REGv2(mScarlet-FLAG) matched positive control in pTwist-CMV | Expresses mScarlet regardless of TDP-43 knockdown. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216162 | TDP-REGv2 Triple Cryptic Cre recombinase in pTwist-CMV | Expresses Cre recombinase in cells lacking TDP-43 function. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216163 | TDP-REGv2 Cryptic PE-Max | Expression of prime editor PEMax (Plasmid #174820) only in cells with TDP-43 loss of function. Note: It is not recommended to produce lentivirus containing TDP-REG sequences – see Depositor Comments |
| 216164 | TDP-REGv2(Gaussia Luciferase)#5 in pTwist-CMV | Expression of secreted Gaussia Luciferase specifically in cells with TDP-43 loss of function. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |
| 216165 | TDP-REGv1(mCherry-FLAG) in pTwist-CMV | Expression of mCherry specifically in cells with TDP-43 knockdown. Uses AARS1-derived cryptic exon to control mCherry expression. Note: It is not recommended to produce lentiviruses containing TDP-REG sequences – see Depositor Comments |