Literature DB >> 19555883

Future applications: gene therapy.

R M Richardson1, V Varenika, J R Forsayeth, K S Bankiewicz.   

Abstract

Gene therapy for brain disorders is one of the most promising frontiers in the practice of restorative neurosurgery. There are significant experimental gene therapy initiatives underway that have led to currently active clinical trials using direct intracerebral delivery of viral vectors, and these treatments have been reported as safe and well tolerated. In the future, other clinical trials will likely use viral vectors to transfer genes that bestow on recipient tissue a desired enzymatic or neurotrophic activity relevant to the treatment of other neurodegenerative diseases, stroke, and traumatic brain injury.

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Year:  2009        PMID： 19555883      PMCID： PMC3030700          DOI： 10.1016/j.nec.2009.04.004

Source DB:  PubMed          Journal:  Neurosurg Clin N Am        ISSN： 1042-3680            Impact factor:   2.509


  15 in total

1.  Real-time visualization and characterization of liposomal delivery into the monkey brain by magnetic resonance imaging.

Authors:  Michal T Krauze; Tracy R Mcknight; Yoji Yamashita; John Bringas; Charles O Noble; Ryuta Saito; Karsten Geletneky; John Forsayeth; Mitchel S Berger; Pamela Jackson; John W Park; Krystof S Bankiewicz
Journal:  Brain Res Brain Res Protoc       Date:  2005-09-21

2.  Effects of the perivascular space on convection-enhanced delivery of liposomes in primate putamen.

Authors:  Michal T Krauze; Ryuta Saito; Charles Noble; John Bringas; John Forsayeth; Tracy R McKnight; John Park; Krystof S Bankiewicz
Journal:  Exp Neurol       Date:  2005-08-16       Impact factor: 5.330

3.  Image-guided, direct convective delivery of glucocerebrosidase for neuronopathic Gaucher disease.

Authors:  R R Lonser; R Schiffman; R A Robison; J A Butman; Z Quezado; M L Walker; P F Morrison; S Walbridge; G J Murray; D M Park; R O Brady; E H Oldfield
Journal:  Neurology       Date:  2006-10-25       Impact factor: 9.910

4.  Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

Authors:  Krystof S Bankiewicz; John Forsayeth; Jamie L Eberling; Rosario Sanchez-Pernaute; Philip Pivirotto; John Bringas; Peter Herscovitch; Richard E Carson; William Eckelman; Bryan Reutter; Janet Cunningham
Journal:  Mol Ther       Date:  2006-07-07       Impact factor: 11.454

5.  Gadolinium-loaded liposomes allow for real-time magnetic resonance imaging of convection-enhanced delivery in the primate brain.

Authors:  Ryuta Saito; Michal T Krauze; John R Bringas; Charles Noble; Tracy R McKnight; Pamela Jackson; Michael F Wendland; Christoph Mamot; Daryl C Drummond; Dimitri B Kirpotin; Keelung Hong; Mitchel S Berger; John W Park; Krystof S Bankiewicz
Journal:  Exp Neurol       Date:  2005-09-28       Impact factor: 5.330

6.  Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease.

Authors:  Steven S Gill; Nikunj K Patel; Gary R Hotton; Karen O'Sullivan; Renée McCarter; Martin Bunnage; David J Brooks; Clive N Svendsen; Peter Heywood
Journal:  Nat Med       Date:  2003-03-31       Impact factor: 53.440

7.  The "perivascular pump" driven by arterial pulsation is a powerful mechanism for the distribution of therapeutic molecules within the brain.

Authors:  Piotr Hadaczek; Yoji Yamashita; Hanna Mirek; Laszlo Tamas; Martha C Bohn; Charles Noble; John W Park; Krystof Bankiewicz
Journal:  Mol Ther       Date:  2006-05-02       Impact factor: 11.454

Review 8.  Gene and cell delivery to the degenerated striatum: status of preclinical efforts in primate models.

Authors:  R Mark Richardson; Paul S Larson; Krystof S Bankiewicz
Journal:  Neurosurgery       Date:  2008-10       Impact factor: 4.654

9.  Convection-enhanced delivery of adeno-associated virus type 2 (AAV2) into the striatum and transport of AAV2 within monkey brain.

Authors:  Piotr Hadaczek; Malgorzata Kohutnicka; Michal T Krauze; John Bringas; Phil Pivirotto; Janet Cunningham; Krystof Bankiewicz
Journal:  Hum Gene Ther       Date:  2006-03       Impact factor: 5.695

10.  Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial.

Authors:  Michael G Kaplitt; Andrew Feigin; Chengke Tang; Helen L Fitzsimons; Paul Mattis; Patricia A Lawlor; Ross J Bland; Deborah Young; Kristin Strybing; David Eidelberg; Matthew J During
Journal:  Lancet       Date:  2007-06-23       Impact factor: 79.321
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  10 in total

1.  Analysis of a simulation algorithm for direct brain drug delivery.

Authors:  Kathryn Hammond Rosenbluth; Jan Felix Eschermann; Gabriele Mittermeyer; Rowena Thomson; Stephan Mittermeyer; Krystof S Bankiewicz
Journal:  Neuroimage       Date:  2011-09-14       Impact factor: 6.556

Review 2.  Tolerogenic dendritic cells and their potential applications.

Authors:  Jim Hu; Yonghong Wan
Journal:  Immunology       Date:  2011-01-05       Impact factor: 7.397

Review 3.  Gene therapy for misfolding protein diseases of the central nervous system.

Authors:  Waldy San Sebastian; Lluis Samaranch; Adrian P Kells; John Forsayeth; Krystof S Bankiewicz
Journal:  Neurotherapeutics       Date:  2013-07       Impact factor: 7.620

4.  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

5.  Cell Therapy From Bench to Bedside Translation in CNS Neurorestoratology Era.

Authors:  Hongyun Huang; Lin Chen; Paul Sanberg
Journal:  Cell Med       Date:  2010-01-01

6.  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 7.  Gene Therapy for Neurodegenerative Diseases.

Authors:  Vivek Sudhakar; R Mark Richardson
Journal:  Neurotherapeutics       Date:  2019-01       Impact factor: 7.620

Review 8.  Technological advances in the surgical treatment of movement disorders.

Authors:  Robert E Gross; Margaret E McDougal
Journal:  Curr Neurol Neurosci Rep       Date:  2013-08       Impact factor: 5.081

Review 9.  Gene Therapy Approach with an Emphasis on Growth Factors: Theoretical and Clinical Outcomes in Neurodegenerative Diseases.

Authors:  Della Grace Thomas Parambi; Khalid Saad Alharbi; Rajesh Kumar; Seetha Harilal; Gaber El-Saber Batiha; Natália Cruz-Martins; Omnia Magdy; Arafa Musa; Dibya Sundar Panda; Bijo Mathew
Journal:  Mol Neurobiol       Date:  2021-10-15       Impact factor: 5.682

Review 10.  Gene-based therapy of Parkinson's Disease: Translation from animal model to human clinical trial employing convection enhanced delivery.

Authors:  Gurwattan S Miranpuri; Lauren Kumbier; Angelica Hinchman; Dominic Schomberg; Anyi Wang; Hope Marshall; Ken Kubota; Chris Ross; Karl Sillay
Journal:  Ann Neurosci       Date:  2012-07
  10 in total

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