Gene therapy tools for brain diseases

S Ingusci, G Verlengia, M Soukupova… - Frontiers in …, 2019 - frontiersin.org
Neurological disorders affecting the central nervous system (CNS) are still incompletely
understood. Many of these disorders lack a cure and are seeking more specific and effective …

Gene therapy approach with an emphasis on growth factors: theoretical and clinical outcomes in neurodegenerative diseases

DGT Parambi, KS Alharbi, R Kumar, S Harilal… - Molecular …, 2022 - Springer
The etiology of many neurological diseases affecting the central nervous system (CNS) is
unknown and still needs more effective and specific therapeutic approaches. Gene therapy …

Viral vectors for therapy of neurologic diseases

SR Choudhury, E Hudry, CA Maguire… - …, 2017 - Elsevier
Neurological disorders–disorders of the brain, spine and associated nerves–are a leading
contributor to global disease burden with a shockingly large associated economic cost …

[HTML][HTML] Gene therapy for the nervous system: challenges and new strategies

CA Maguire, SH Ramirez, SF Merkel, M Sena-Esteves… - …, 2014 - Elsevier
Current clinical treatments for central nervous system (CNS) diseases, such as Parkinson's
disease and glioblastoma do not halt disease progression and have significant treatment …

Current status and challenges associated with CNS-targeted gene delivery across the BBB

S Kimura, H Harashima - Pharmaceutics, 2020 - mdpi.com
The era of the aging society has arrived, and this is accompanied by an increase in the
absolute numbers of patients with neurological disorders, such as Alzheimer's disease (AD) …

Gene therapy in the CNS

LC Costantini, JC Bakowska, XO Breakefield… - Gene therapy, 2000 - nature.com
Gene therapy for neurological disorder is currently an experimental concept. The goals for
clinical utilization are the relief of symptoms, slowing of disease progression, and correction …

Clinical applications involving CNS gene transfer

B Kantor, T McCown, P Leone, SJ Gray - Advances in genetics, 2014 - Elsevier
Diseases of the central nervous system (CNS) have traditionally been the most difficult to
treat by traditional pharmacological methods, due mostly to the blood–brain barrier and the …

Gene therapy of the brain: the trans-vascular approach

F Schlachetzki, Y Zhang, RJ Boado, WM Pardridge - Neurology, 2004 - AAN Enterprises
Many chronic neurologic diseases do not respond to small molecule therapeutics, and have
no effective long-term therapy. Gene therapy offers the promise of an effective cure for both …

Applications of gene therapy to the CNS

U Blömer, L Naldini, IM Verma, D Trono… - Human molecular …, 1996 - academic.oup.com
Gene therapy is a new method with potential for treating a broad range of acquired and
inherited neurologic diseases, where the causative gene defect or deletion has been …

In pursuit of new developments for gene therapy of human diseases

G Palu, R Bonaguro, A Marcello - Journal of biotechnology, 1999 - Elsevier
Gene therapy aims at transferring a therapeutic gene into human somatic cells in order to
treat a disease. Originally addressed to hereditary genetic disorders, gene therapy has …