» Articles » PMID: 34366372

Improved Delivery Methods for Gene Therapy and Cell Transplantation in Parkinson's Disease

Overview
Publisher Sage Publications
Specialty Neurology
Date 2021 Aug 9
PMID 34366372
Citations 3
Authors
Affiliations
Soon will be listed here.
Abstract

A number of cell transplantation and gene therapy trials have been performed over the last three decades in an effort to restore function in Parkinson's disease. Much has been learned about optimizing delivery methods for these therapeutics. This is particularly true in gene therapy, which has predominated the clinical trial landscape in recent years; however, cell transplantation for Parkinson's disease is currently undergoing a renaissance. Innovations such as cannula design, iMRI-guided surgery and an evolution in delivery strategy has radically changed the way investigators approach clinical trial design. Future therapeutic strategies may employ newer delivery methods such as chronically implanted infusion devices and focal opening of the blood brain barrier with focused ultrasound.

Citing Articles

Advancements in surgical treatments for Huntington disease: From pallidotomy to experimental therapies.

Kim L, Kundu B, Moretti P, Lozano A, Rahimpour S Neurotherapeutics. 2024; 21(6):e00452.

PMID: 39304438 PMC: 11585891. DOI: 10.1016/j.neurot.2024.e00452.


Preclinical evaluation of transaxial intraputaminal trajectory for enhanced distribution of grafted cells in Parkinson's disease.

Emborg M, Mancinelli A, Colwell J, Zinnen A, Pape B, Brunner K J Neurosurg. 2024; 141(6):1554-1566.

PMID: 39059426 PMC: 11609033. DOI: 10.3171/2024.4.JNS24367.


Genetically engineered mesenchymal stem cells with dopamine synthesis for Parkinson's disease in animal models.

Li J, Li N, Wei J, Feng C, Chen Y, Chen T NPJ Parkinsons Dis. 2022; 8(1):175.

PMID: 36550118 PMC: 9780305. DOI: 10.1038/s41531-022-00440-6.

References
1.
Kanda T, Ishii K, Kawaguchi H, Kitajima K, Takenaka D . High signal intensity in the dentate nucleus and globus pallidus on unenhanced T1-weighted MR images: relationship with increasing cumulative dose of a gadolinium-based contrast material. Radiology. 2014; 270(3):834-41. DOI: 10.1148/radiol.13131669. View

2.
Alarcon-Aris D, Pavia-Collado R, Miquel-Rio L, Coppola-Segovia V, Ferres-Coy A, Ruiz-Bronchal E . Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys. EBioMedicine. 2020; 59:102944. PMC: 7452525. DOI: 10.1016/j.ebiom.2020.102944. View

3.
McMahon D, OReilly M, Hynynen K . Therapeutic Agent Delivery Across the Blood-Brain Barrier Using Focused Ultrasound. Annu Rev Biomed Eng. 2021; 23:89-113. DOI: 10.1146/annurev-bioeng-062117-121238. View

4.
Marks Jr W, Ostrem J, Verhagen L, Starr P, Larson P, Bakay R . 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. 2008; 7(5):400-8. DOI: 10.1016/S1474-4422(08)70065-6. View

5.
Lindvall O, Widner H, Rehncrona S, Brundin P, Odin P, Gustavii B . Transplantation of fetal dopamine neurons in Parkinson's disease: one-year clinical and neurophysiological observations in two patients with putaminal implants. Ann Neurol. 1992; 31(2):155-65. DOI: 10.1002/ana.410310206. View