Leveraging Adeno-Associated Viruses (AAVs) in therapeutic approaches for monogenic neurological diseases

Supplied by PerkinElmer on Wednesday, 07 September, 2022


Within the field of neurological diseases, there is great interest in looking at rare diseases of monogenic origin with the hope of developing disease-modifying gene therapies, as opposed to treatments for symptom management. Therefore, using relatively tunable systems like recombinant AAVs, scientists are also exploring in vivo gene delivery in parallel to ex vivo.

Learn about key strategies and innovations in this space that are enabling advanced gene therapy.


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