Archive 1995–2023
2012, volume 28, issue 1
Review article
Adeno-associated virus vectors in gene therapies of neurodegenerative diseases
Adriana Wawer1,
Anna Sznejder-Pachołek1,
Ilona Joniec-Maciejak1,
Joanna Schwenkgrub1,
Andrzej Członkowski1
1. Katedra i Zakład Farmakologii Doświadczalnej i Klinicznej w Warszawie
Farmakoterapia w Psychiatrii i Neurologii, 2012, 1, 33–40
Keywords: AAV vectors, gene therapy, neurodegenerative diseases, Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis
Summary
The search for effective therapy of neurodegenerative diseases is an important issue and an important challenge. Nowadays, there are high hopes for the gene therapy based treatment. This involves using a noninfectious virus administered directly into the brain. AAV vectors are increasingly being used as tools for delivery of therapeutic agents to the CNS. The present paper aims to review clinical trials carried out so far with the use of the AAV vectors in treatment of neurodegenerative disorders: Parkinson’s disease, Alzheimer’s disease, amyotrophic lateral sclerosis, Canavan disease and late infantile neuronal ceroid lipofuscinosis. Results obtained from these trials show good tolerance of AAV vectors in the CNS and safety of therapies.
Correspondence address
Ilona Joniec-Maciejak,
Katedra i Zakład Farmakologii Doświadczalnej i Klinicznej,
ul. Krakowskie Przedmieście 26/28, 00-927 Warszawa,
tel./fax 22 826 21 16,
e-mail: [email]ilona.joniec@wum.edu.pl[/email]
Katedra i Zakład Farmakologii Doświadczalnej i Klinicznej,
ul. Krakowskie Przedmieście 26/28, 00-927 Warszawa,
tel./fax 22 826 21 16,
e-mail: [email]ilona.joniec@wum.edu.pl[/email]