Literature DB >> 19254173

Functional effects of AAV2-GDNF on the dopaminergic nigrostriatal pathway in parkinsonian rhesus monkeys.

Jamie L Eberling1, Adrian P Kells, Philip Pivirotto, Janine Beyer, John Bringas, Howard J Federoff, John Forsayeth, Krystof S Bankiewicz.   

Abstract

We investigated the safety and neuroregenerative potential of an adeno-associated virus (AAV2) containing human glial cell line-derived neurotrophic factor (GDNF) in an MPTP primate model of Parkinson's disease. Dopaminergic function was evaluated by positron emission tomography with 6-[(18)F]fluoro-l-m-tyrosine (FMT) before and after AAV2-GDNF or phosphate-buffered saline infusion bilaterally into the putamen. FMT uptake was significantly increased bilaterally in the putamen of AAV2-GDNF but not phosphate-buffered saline-treated animals 6 months after infusion, indicating increased dopaminergic activity in the nigrostriatal pathways. AAV2-GDNF-treated animals also showed clinical improvement without adverse effects. These findings are consistent with our previous report in aged nonhuman primates that showed evidence of enhanced use of striatal dopamine and dopaminergic nigrostriatal innervation. Clinical improvement and evidence of functional recovery in the nigrostriatal pathway, and the absence of adverse effects, support the safety of this approach for the delivery of GDNF over a 6-month period.

Entities:  

Mesh:

Substances:

Year:  2009        PMID: 19254173      PMCID: PMC2725183          DOI: 10.1089/hum.2008.201

Source DB:  PubMed          Journal:  Hum Gene Ther        ISSN: 1043-0342            Impact factor:   5.695


  42 in total

1.  Research on Parkinson disease.

Authors:  John T Slevin; Greg A Gerhardt; Charles D Smith; Don M Gash; Richard Kryscio; Byron Young
Journal:  J Neurosurg       Date:  2005-02       Impact factor: 5.115

2.  Six-month continuous intraputamenal infusion toxicity study of recombinant methionyl human glial cell line-derived neurotrophic factor (r-metHuGDNF in rhesus monkeys.

Authors:  David N Hovland; Robert B Boyd; Mark T Butt; Jeffery A Engelhardt; Michael S Moxness; Mark H Ma; Maurice G Emery; Nadia B Ernst; Randall P Reed; Jillynne R Zeller; Don M Gash; Donna M Masterman; Beth M Potter; Mary E Cosenza; Ruth M Lightfoot
Journal:  Toxicol Pathol       Date:  2007-12       Impact factor: 1.902

3.  Towards gene therapy for Parkinson's disease.

Authors:  Stéphane Palfi
Journal:  Lancet Neurol       Date:  2008-04-02       Impact factor: 44.182

4.  Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach.

Authors:  K S Bankiewicz; J L Eberling; M Kohutnicka; W Jagust; P Pivirotto; J Bringas; J Cunningham; T F Budinger; J Harvey-White
Journal:  Exp Neurol       Date:  2000-07       Impact factor: 5.330

5.  Delivery of neurturin by AAV2 (CERE-120)-mediated gene transfer provides structural and functional neuroprotection and neurorestoration in MPTP-treated monkeys.

Authors:  Jeffrey H Kordower; Christopher D Herzog; Biplob Dass; Roy A E Bakay; James Stansell; Mehdi Gasmi; Raymond T Bartus
Journal:  Ann Neurol       Date:  2006-12       Impact factor: 10.422

6.  Clinicopathological findings following intraventricular glial-derived neurotrophic factor treatment in a patient with Parkinson's disease.

Authors:  J H Kordower; S Palfi; E Y Chen; S Y Ma; T Sendera; E J Cochran; E J Cochran; E J Mufson; R Penn; C G Goetz; C D Comella
Journal:  Ann Neurol       Date:  1999-09       Impact factor: 10.422

Review 7.  Intracranial delivery of proteins and peptides as a therapy for neurodegenerative diseases.

Authors:  Richard Grondin; Zhiming Zhang; Yi Ai; Don M Gash; Greg A Gerhardt
Journal:  Prog Drug Res       Date:  2003

8.  Biodistribution of adeno-associated virus type-2 in nonhuman primates after convection-enhanced delivery to brain.

Authors:  Janet Cunningham; Philip Pivirotto; John Bringas; Brian Suzuki; Sharmila Vijay; Laura Sanftner; Marina Kitamura; Curtis Chan; Krystof S Bankiewicz
Journal:  Mol Ther       Date:  2008-06-03       Impact factor: 11.454

9.  Hemiparkinsonism in monkeys after unilateral internal carotid artery infusion of 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP).

Authors:  K S Bankiewicz; E H Oldfield; C C Chiueh; J L Doppman; D M Jacobowitz; I J Kopin
Journal:  Life Sci       Date:  1986-07-07       Impact factor: 5.037

10.  Striatal delivery of CERE-120, an AAV2 vector encoding human neurturin, enhances activity of the dopaminergic nigrostriatal system in aged monkeys.

Authors:  Christopher D Herzog; Biplob Dass; James E Holden; James Stansell; Mehdi Gasmi; Mark H Tuszynski; Raymond T Bartus; Jeffrey H Kordower
Journal:  Mov Disord       Date:  2007-06-15       Impact factor: 10.338

View more
  48 in total

Review 1.  Neurorestoration.

Authors:  Mikko Airavaara; Merja H Voutilainen; Yun Wang; Barry Hoffer
Journal:  Parkinsonism Relat Disord       Date:  2012-01       Impact factor: 4.891

2.  Is GDNF beneficial in Parkinson disease?

Authors:  Barry J Hoffer; Brandon K Harvey
Journal:  Nat Rev Neurol       Date:  2011-09-20       Impact factor: 42.937

3.  Transgene expression in the striatum following intracerebral injections of DNA nanoparticles encoding for human glial cell line-derived neurotrophic factor.

Authors:  A M Fletcher; T H Kowalczyk; L Padegimas; M J Cooper; D M Yurek
Journal:  Neuroscience       Date:  2011-08-04       Impact factor: 3.590

Review 4.  Cellular and Molecular Aspects of Parkinson Treatment: Future Therapeutic Perspectives.

Authors:  Khosro Jamebozorgi; Eskandar Taghizadeh; Daryoush Rostami; Hosein Pormasoumi; George E Barreto; Seyed Mohammad Gheibi Hayat; Amirhossein Sahebkar
Journal:  Mol Neurobiol       Date:  2018-11-05       Impact factor: 5.590

Review 5.  Viral vectors for neurotrophic factor delivery: a gene therapy approach for neurodegenerative diseases of the CNS.

Authors:  Seung T Lim; Mikko Airavaara; Brandon K Harvey
Journal:  Pharmacol Res       Date:  2009-10-17       Impact factor: 7.658

6.  Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain.

Authors:  R Mark Richardson; Adrian P Kells; Alastair J Martin; Paul S Larson; Philip A Starr; Peter G Piferi; Geoffrey Bates; Lisa Tansey; Kathryn H Rosenbluth; John R Bringas; Mitchel S Berger; Krystof S Bankiewicz
Journal:  Stereotact Funct Neurosurg       Date:  2011-04-14       Impact factor: 1.875

7.  Interventional MRI-guided putaminal delivery of AAV2-GDNF for a planned clinical trial in Parkinson's disease.

Authors:  R Mark Richardson; Adrian P Kells; Kathryn H Rosenbluth; Ernesto Aguilar Salegio; Massimo S Fiandaca; Paul S Larson; Philip A Starr; Alastair J Martin; Russell R Lonser; Howard J Federoff; John R Forsayeth; Krystof S Bankiewicz
Journal:  Mol Ther       Date:  2011-02-22       Impact factor: 11.454

Review 8.  Cell replacement therapy is the remedial solution for treating Parkinson's disease.

Authors:  Venkatesan Dhivya; Vellingiri Balachandar
Journal:  Stem Cell Investig       Date:  2017-06-30

9.  Targeted gene transfer to the brain via the delivery of brain-penetrating DNA nanoparticles with focused ultrasound.

Authors:  Brian P Mead; Panagiotis Mastorakos; Jung Soo Suk; Alexander L Klibanov; Justin Hanes; Richard J Price
Journal:  J Control Release       Date:  2015-12-28       Impact factor: 9.776

10.  Autologous mesenchymal stem cell-derived dopaminergic neurons function in parkinsonian macaques.

Authors:  Takuya Hayashi; Shohei Wakao; Masaaki Kitada; Takayuki Ose; Hiroshi Watabe; Yasumasa Kuroda; Kanae Mitsunaga; Dai Matsuse; Taeko Shigemoto; Akihito Ito; Hironobu Ikeda; Hidenao Fukuyama; Hirotaka Onoe; Yasuhiko Tabata; Mari Dezawa
Journal:  J Clin Invest       Date:  2012-12-03       Impact factor: 14.808

View more

北京卡尤迪生物科技股份有限公司 © 2022-2023.