Challenges for Gene Therapy of CNS Disorders and Implications for Parkinson's Disease Therapies

Abstract

Get full access to this article

View all access options for this article.

References

Asokan

, Schaffer

D.V.

, Samulski

R.J.

2012. The AAV vector toolkit: Poised at the clinical crossroads. Mol. Ther.(in press).

Bell

C.L.

, Vandenberghe

L.H.

, Bell

et al. 2011. The AAV9 receptor and its modification to improve in vivo lung gene transfer in mice. J. Clin. Invest., 121:2427–2435.

Boutin

, Monteilhet

, Veron

et al. 2010. Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: Implications for gene therapy using AAV vectors. Hum. Gene Ther., 21:704–712.

Calcedo

, Morizono

, Wang

et al. 2011. Adeno-associated virus antibody profiles in newborns, children, and adolescents. Clin. Vaccine Immunol., 18:1586–1588.

Christine

C.W.

, Starr

P.A.

, Larson

P.S.

et al. 2009. Safety and tolerability of putaminal AADC gene therapy for Parkinson disease. Neurology, 73:1662–1669.

Diederich

N.J.

, Goetz

C.G.

2008. The placebo treatments in neurosciences: New insights from clinical and neuroimaging studies. Neurology, 71:677–684.

Drinkut

, Tereshchenko

, Schulz

J.B.

et al. 2011. Efficient gene therapy for Parkinson's disease using astrocytes as hosts for localized neurotrophic factor delivery. Mol. Ther., 20:534–543.

Duque

, Joussemet

, Riviere

et al. 2009. Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons. Mol. Ther., 17:1187–1196.

Federici

, Taub

J.S.

, Baum

G.R.

et al. 2011. Robust spinal motor neuron transduction following intrathecal delivery of AAV9 in pigs. Gene Ther.(in press).

10.

Forsayeth

J.R.

, Bankiewicz

K.S.

2011. AAV9: Over the fence and into the woods. Mol. Ther., 19:1006–1007.

11.

Foust

K.D.

, Nurre

, Montgomery

C.L.

et al. 2009. Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes. Nat. Biotechnol., 27:59–65.

12.

Goetz

C.G.

, Wuu

, McDermott

M.P.

et al. 2008. Placebo response in Parkinson's disease: Comparisons among 11 trials covering medical and surgical interventions. Mov. Disord., 23:690–699.

13.

Gray

S.J.

, Blake

B.L.

, Criswell

H.E.

et al. 2010. Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood–brain barrier (BBB) Mol. Ther., 18:570–578.

14.

Gray

S.J.

, Foti

S.B.

, Schwartz

J.W.

et al. 2011a. Optimizing promoters for recombinant adeno-associated virus-mediated gene expression in the peripheral and central nervous system using self-complementary vectors. Hum. Gene Ther., 22:1143–1153.

15.

Gray

S.J.

, Matagne

, Bachaboina

et al. 2011b. Preclinical differences of intravascular AAV9 delivery to neurons and glia: A comparative study of adult mice and nonhuman primates. Mol. Ther., 19:1058–1069.

16.

Ichinose

, Ohye

, Fujita

et al. 1994. Quantification of mRNA of tyrosine hydroxylase and aromatic l-amino acid decarboxylase in the substantia nigra in Parkinson's disease and schizophrenia. J. Neural Transm. Park. Dis. Dement., 8:149–158.

17.

Jellinger

K.A.

2012. Neuropathology of sporadic Parkinson's disease: Evaluation and changes of concepts. Mov. Disord, 27:8–30.

18.

Kugler

, Lingor

, Scholl

et al. 2003. Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units. Virology, 311:89–95.

19.

LeWitt

P.A.

, Rezai

A.R.

, Leehey

M.A.

et al. 2011. AAV2-GAD gene therapy for advanced Parkinson's disease: A double-blind, sham-surgery controlled, randomised trial. Lancet Neurol., 10:309–319.

20.

Manfredsson

F.P.

, Rising

A.C.

, Mandel

R.J.

2009. AAV9: A potential blood–brain barrier buster. Mol. Ther., 17:403–405.

21.

Manno

C.S.

, Pierce

G.F.

, Arruda

V.R.

et al. 2006. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med., 12:342–347.

22.

Marks

W.J.

Jr. , Ostrem

J.L.

, Verhagen

et al. 2008. Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: An open-label, phase I trial. Lancet Neurol., 7:400–408.

23.

Marks

Jr. , Bartus

, Siffert

et al. 2010. Gene delivery of AAV2-neurturin for Parkinson's disease: A double-blind, randomised, controlled trial. Lancet Neurol., 9:1164–1172.

24.

Martinez-Martin

2011. The importance of non-motor disturbances to quality of life in Parkinson's disease. J. Neurol. Sci., 310:12–16.

25.

Mattar

C.N.

, Waddington

S.N.

, Biswas

et al. 2012. Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems. Gene Ther.(in press).

26.

McCown

T.J.

2011. Adeno-associated virus (AAV) vectors in the CNS. Curr. Gene Ther., 11:181–188.

27.

Mittermeyer

, Christine

C.W.

, Rosenbluth

K.H.

et al. 2012. Long-term evaluation of a phase I study of AADC gene therapy for Parkinson's disease. Hum. Gene Ther.(this issue).

28.

Obeso

J.A.

, Olanow

C.W.

, Nutt

J.G.

2000. Levodopa motor complications in Parkinson's disease. Trends Neurosci., 23:S2–S7.

29.

Pulicherla

, Shen

, Yadav

et al. 2011. Engineering liver-detargeted AAV9 vectors for cardiac and musculoskeletal gene transfer. Mol. Ther., 19:1070–1078.

30.

Samaranch

, Salegio

E.A.

, San Sebastian

et al. 2012. AAV9 transduction in the central nervous system of nonhuman primates. Hum. Gene Ther.(this issue).

31.

Shen

, Bryant

K.D.

, Brown

S.M.

et al. 2011. Terminal N-linked galactose is the primary receptor for adeno-associated virus 9. J. Biol. Chem., 286:13532–13540.

32.

Witt

, Marks

W.J.

Jr.

2011. An update on gene therapy in Parkinson's disease. Curr. Neurol. Neurosci. Rep., 11:362–370.

33.

Xie

, Xie

, Zhang

et al. 2011. MicroRNA-regulated, systemically delivered rAAV9: A step closer to CNS-restricted transgene expression. Mol. Ther., 19:526–535.

34.

Zhang

, Yang

, Mu

et al. 2011. Several rAAV vectors efficiently cross the blood–brain barrier and transduce neurons and astrocytes in the neonatal mouse central nervous system. Mol. Ther., 19:1440–1448.