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CLN2 Mechanism of Disease 

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ADA-SCID Mechanism of Disease

medical illustration of This series of panels shows the comparison of the breakdown of cellular waste in healthy cells vs cells with ceroid lipofuscinosis type 2 (CLN2). The bottom panel shows the MOA of the NAV technology, which uses an AAV to transport functional TPP1 into the CLN2 patient.
Next medical illustration of This molecular biology illustration shows a three-way comparison of the FGFR3 signalling pathway that regulates bone growth in normal, achondroplasia, and achondroplasia treated with TA-46. TA-46 is an investigational, soluble recombinant FGFR3 decoy in clinical trials by Therachon / Pfizer.

Mechanism of Action of TA-46 in Achondroplasia

This series of panels shows the comparison of the breakdown of cellular waste in healthy cells vs cells with ceroid lipofuscinosis type 2 (CLN2). The bottom panel shows the MOA of the NAV technology, which uses an AAV to transport functional TPP1 into the CLN2 patient.

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© Diana Kryski