| | |  | | | | Record from Web of Science® | | | | | | |
| LONG-TERM GENE-EXPRESSION AND PHENOTYPIC CORRECTION USING ADENOASSOCIATED VIRUS VECTORS IN THE MAMMALIAN BRAIN |
|
|
| Author(s): KAPLITT MG, LEONE P, SAMULSKI RJ, XIAO X, PFAFF DW, OMALLEY KL, DURING MJ |
| Source: NATURE GENETICS Volume: 8 Issue: 2 Pages: 148-154 Published: OCT 1994 |
| Times Cited: 681 References: 43 |
| Abstract: Adeno-associated viral (AAV) vectors are non-pathogenic, integrating DNA vectors in which all viral genes are removed and helper virus is completely eliminated. To evaluate this system in the post-mitotic cells of the brain, we found that an AAV vector containing the lacZ gene (AAVlac) resulted in expression of P-galactosidase up to three months post-injection in vivo. A second vector expressing human tyrosine hydroxylase (AAVth) was injected into the denervated striatum of unilateral 6-hydroxydopamine-lesioned rats. Tyrosine hydroxylase (TH) immunoreactivity was detectable in striatal neurons and glia for up to four months and we also found significant behavioural recovery in lesioned rats treated with AAVth versus AAVlac controls. Safe and stable TH gene transfer into the denervated striatum may have potential for the genetic therapy of Parkinson's disease. |
| Document Type: Article |
| Language: English |
Addresses:
1. YALE UNIV, SCH MED, DEPT SURG, MOLEC PHARMACOL & NEUROGENET LAB, NEW HAVEN, CT 06520 USA
2. ROCKEFELLER UNIV, NEUROBIOL & BEHAV LAB, NEW YORK, NY 10021 USA
3. UNIV N CAROLINA, CTR GENE THERAPY, CHAPEL HILL, NC 27599 USA
4. MERLIN PHARMACEUT, NEW YORK, NY 10014 USA
5. WASHINGTON UNIV, SCH MED, DEPT ANAT & NEUROBIOL, ST LOUIS, MO 63110 USA |
| Publisher: NATURE PUBLISHING CO, 345 PARK AVE SOUTH, NEW YORK, NY 10010-1707 |
| Subject Category: Genetics & Heredity |
| IDS Number: PL123 |
| ISSN: 1061-4036 |
|
| | | | | | | | | Record from Web of Science® | | | | | | | | | |