Disease Resources

A list of rare diseases and how Gene Therapy can play a role within the disease.

What is Krabbe Disease?

 

krabbe infographic final 03sep19

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What is Gene Therapy for GM1 Gangliosidosis?

gm1 infographic final 10sep19

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Crigler-Najjar and the Potential for Gene Therapy

crigler najjar infographic

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Maple Syrup Urine Disease (MSUD)

 

msud infographic 01

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What is Gene Therapy for MPS I?

 

Intrathecal Gene Therapy for MPS I summary can be found here

mps i infographic final 27mar2018

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HoFH and the Potential for Gene Therapy

Gene Therapy for HoFH summary can be found here

hofh infographic final 27mar2018

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