• Parkinson's Disease AAV Research Tools


    MJFF Image

    Vigene has partnered with The Michael J. Fox Foundation for Parkinson’s Research (MJFF) to distribute viral vectors targeting alpha-synuclein (aSyn), a major protein implicated in the pathology of Parkinson’s disease.
    Learn more about aSyn, Parkinson's disease, and MJFF below.

    alpha-synuclein AAV viral vectors

    aSyn knockdown: Viral vectors expressing a miR to specifically knockdown mouse aSyn, human aSyn, or a scrambled control, as well as a GFP reporter to measure transduction efficiency.

    aSyn overexpression (coming 2019): Two different AAV pseudotypes to overexpress wildtype human aSyn—rAAV2/2 aSyn and rAAV2/5 aSyn—with rAAV2/2 BFP and rAAV2/5 BFP as their respective controls.

    alpha-synuclein knockdown viral vectors

    In collaboration with MJFFs Parkinson's Disease Research Tools Consortium, three AAV viral vectors were generated for preclinical use that knockdown the levels of aSyn. These viral vectors each express a miR to specifically knockdown mouse aSyn, human aSyn, or a scrambled control, as well as a GFP reporter to measure transduction efficiency. With the help of MJFF industry partners, these vectors are fully validated for expression and knockdown efficiency in vitro as well as in vivo. View validation data.

    Order alpha-synuclein AAV tools

    Cat #

    aSyn

    AAV Vector

    Titer

    Knockdown (in vitro; in vivo)

    Price

    Download

    GD1009-RV-H

    human

    AAV1/2-CAG-Human-SNCA-3xmiR/GFP-WPRE-BGH-polyA

    0.5x10^12 vg/mL; 100uL

    ~75%;

    ~100%

    $250

    datasheet or
    NTI sequence

    GD1009-RV-M

    mouse

    AAV1/2-CAG-Mouse-SNCA-3xmiR/GFP-WPRE-BGH-polyA

    0.5x10^12 vg/mL; 100uL

    ~75%;

    ~100%

    $250

    datasheet or
    NTI sequence

    GD1009-RV-C

    scrambled control

    AAV1/2-CAG-Scrambled-Control-3xSCRmiR/GFP-WPRE-BGH-polyA

    0.5x10^12 vg/mL; 100uL

    ~75%;

    ~100%

    $250

    datasheet or
    NTI sequence

    Recommended Dose

    For in vitro use, it is recommended to start with a 1:100 dilution of the stock solution supplied at 0.5 x 10 12 vg/mL. Dilute further as necessary. Wait 2-3 days for knockdown in HEK293 cells and 7-9 days for knockdown in primary neurons.

    For in vivo use, it is recommended to start with a 2 µL injection of the undiluted stock solution supplied at 0.5 x 10 12 vg/mL. Dilute further as necessary. It is generally recommended to use a 3 week post-injection interval. Doses and recommended duration of expression will vary based on cell type, model, and desired degree of knockdown.

    Alpha-synuclein overexpression viral vectors

    Ready to ship mid-late 2019. More information coming soon.

    Alpha–synuclein

    Alpha-synuclein (aSyn) is a protein whose function in the healthy brain is currently unknown. aSyn is of great interest to Parkinson's researchers because it is a major constituent of Lewy bodies, which are protein clumps that are the pathological hallmark of Parkinson's disease. There is compelling evidence from recent studies that aSyn may play a role in the development of both familial (rare) and sporadic (more common) cases of Parkinson's disease.

    Alpha–synuclein therapies in clinical trials

    Currently, five companies (Prothena-Roche, Biogen, Neuropore, Proclara, and AFFiRiS) have ongoing clinical trials targeting aSyn with even more companies in the pre-clinical phase. MJFF funding has helped support both Proclara and AFFiRiS in their preclinical and Phase I trials. The latter company, the Austrian biotech AFFiRiS, uses a vaccine of synthetic protein to elicit antibodies that neutralize aSyn and clear it from the brain. Click here for more information on the ongoing clinical trials.

    The Michael J. Fox Foundation Research Tools Consortium

    To speed up Parkinson’s research and accelerate therapeutic development, MJFF invests in the generation and availability of critical tools for Parkinson’s disease investigators. MJFF has a multi-faceted approach to developing these research tools. MJFF identifies critical needs through discussions with key scientific experts, generates tools through partnerships with leading experts and companies, evaluates these resources for quality control, and distributes the tools broadly through vendors and partner organizations. More information on MJFF tools and partnerships and can be found in the MJFF Research Tools Catalog.

    Additional Viral Resources

    Ordering Instructions: Shipping and payment information
    Product Manuals: How to use your virus
    FAQs : Answers to commonly asked questions
    Publications: showcasing Vigene products and services
    Recipient instructions: information on viral safety and storage