To save researchers time and resources, The Michael J. Fox Foundation has made a number of tools available to the scientific community at low cost, with rapid delivery.
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Tools Consortium
MJFF is working with industry to develop priority tools.
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Preclinical Models
Learn more about the various in vivo models used in Parkinson’s disease research.
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KOLF2.1J human iPSC line with CRISPR-engineered mutations in ATP13A2, including:
- -T517I (heterozygous and homozygous)
- -R745H (heterozygous and homozygous) - in development, est late 2026
- -M854R (heterozygous and homozygous)
- -c1306 (heterozygous and homozygous)
- -DOX-NGN2 + TMEM192-HA + c1306 (heterozygous and homozygous) - in development, est late 2026
These lines were generated within the iPSC Neurodegeneration Initiative for Parkinson’s Disease (iNDI-PD). This cell line development project is is supported by the Aligning Science Across Parkinson’s (ASAP) Initiative and the MJFF Targets to Therapies (T2T) Initiative.
Estimated Availability: Late 2026
KOLF2.1J human iPSC line with CRISPR-engineered mutations in LRRK2, including:
- -R1298H (heterozygous and homozygous) - in development, est late 2025
- -R1441C (heterozygous and homozygous)
- -R1441G (heterozygous and homozygous)
- -R1628H (heterozygous and homozygous) - in development, est late 2025
- -Y1699C (heterozygous and homozygous)
- -L1795F (heterozygous and homozygous) - in development, est late 2025
- -G2019S (heterozygous and homozygous)
- -I2020T (heterozygous and homozygous)
- -N2081D (heterozygous and homozygous)
- -G2385R (heterozygous and homozygous) - in development, est late 2026
These lines were generated within the iPSC Neurodegeneration Initiative for Parkinson’s Disease (iNDI-PD) which is supported by the Aligning Science Across Parkinson’s (ASAP) Initiative.
Estimated Availability: Late 2025
Mouse expressing either nuclear localized SNCAWT:TagBFP, SNCAA30P:mTFP1, or SNCAA53T:mKO2 in cre-expressing tissues of the offspring directed by CAG promoter followed by three pairs of orthogonal lox sites (LoxN, Lox2272, and LoxP), and the SNCAbow construct; all targeted into the Gt(ROSA)26Sor locus. Each α-synuclein sequence contains a C-terminal epitope tag: SNCAWT tagged with V5, SNCAA30P tagged with 3XHA, or SNCAA53T tagged with Myc. Model was generated by Rodger Liddle at Duke University Medical Center and deposited through the MJFF Sponsored Tools Program as part of the Aligning Science Across Parkinson’s (ASAP) Initiative. RRID:IMSR_JAX:037603
KOLF2.1J human iPSC line with CRISPR-engineered mutations in GBA1, including:
- -K198E (heterozygous and homozygous)
- -E326K (heterozygous and homozygous)
- -T369M (heterozygous and homozygous)
- -N370S (heterozygous and homozygous)
- -L444P (heterozygous and homozygous) - in development, est late 2025
- -D448H (heterozygous and homozygous) - in development, est late 2025
- -D448V (heterozygous and homozygous)
- -Noncoding rs3115534 (heterozygous and homozygous) - in development, est late 2025
- -DOX-NGN2 + TMEM192-HA + L444P (heterozygous and homozygous) - in development, est late 2026
These lines were generated within the iPSC Neurodegeneration Initiative for Parkinson’s Disease (iNDI-PD) which is supported by the Aligning Science Across Parkinson’s (ASAP) Initiative. Cell line development is also supported by MJFF Targets to Therapies (T2T) Initiative.
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