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Gene therapy aims to treat the genetic disorders by replacing or inactivating genes or introducing corrective genes into cells1
Find out more about gene therapy and the future on AADC deficiency management:
References: 1. American Society of Gene and Cell Therapy. Available at: https://patienteducation.asgct.org/gene-therapy-101/gene-therapybasics.Last accessed October 2021
This website is provided for healthcare professionals residing in the Middle East and Africa.
This educational website provides information to support the early and accurate diagnosis of rare neurotransmitter disorders, including Aromatic L-amino Acid Decarboxylase (AADC) deficiency.