Various AAV serotypes and their applications in gene therapy: an overview

SS Issa, AA Shaimardanova, VV Solovyeva… - Cells, 2023 - mdpi.com
Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

BDNF as a promising therapeutic agent in Parkinson's disease

E Palasz, A Wysocka, A Gasiorowska… - International journal of …, 2020 - mdpi.com
Brain-derived neurotrophic factor (BDNF) promotes neuroprotection and neuroregeneration.
In animal models of Parkinson's disease (PD), BDNF enhances the survival of dopaminergic …

Gene therapy comes of age

CE Dunbar, KA High, JK Joung, DB Kohn, K Ozawa… - Science, 2018 - science.org
BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human …

[HTML][HTML] AAV vectors applied to the treatment of CNS disorders: Clinical status and challenges

L Kang, S Jin, J Wang, Z Lv, C Xin, C Tan… - Journal of Controlled …, 2023 - Elsevier
In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Mitochondria and lipid peroxidation in the mechanism of neurodegeneration: Finding ways for prevention

PR Angelova, N Esteras… - Medicinal Research …, 2021 - Wiley Online Library
The world's population aging progression renders age‐related neurodegenerative diseases
to be one of the biggest unsolved problems of modern society. Despite the progress in …

Therapeutic AAV gene transfer to the nervous system: a clinical reality

E Hudry, LH Vandenberghe - Neuron, 2019 - cell.com
Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

[HTML][HTML] Parkinson's disease and its management: part 1: disease entity, risk factors, pathophysiology, clinical presentation, and diagnosis

G DeMaagd, A Philip - Pharmacy and therapeutics, 2015 - ncbi.nlm.nih.gov
Parkinson’s Disease and Its Management - PMC Back to Top Skip to main content NIH NLM
Logo Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation Search …

Emerging therapies in Parkinson disease—repurposed drugs and new approaches

A Elkouzi, V Vedam-Mai, RS Eisinger… - Nature Reviews …, 2019 - nature.com
Parkinson disease (PD) treatment options have conventionally focused on dopamine
replacement and provision of symptomatic relief. Current treatments cause undesirable …

Tet-on systems for doxycycline-inducible gene expression

AT Das, L Tenenbaum, B Berkhout - Current gene therapy, 2016 - ingentaconnect.com
The tetracycline-controlled Tet-Off and Tet-On gene expression systems are used to regulate
the activity of genes in eukaryotic cells in diverse settings, varying from basic biological …

Randomized trial of intermittent intraputamenal glial cell line-derived neurotrophic factor in Parkinson's disease

A Whone, M Luz, M Boca, M Woolley, L Mooney… - Brain, 2019 - academic.oup.com
We investigated the effects of glial cell line-derived neurotrophic factor (GDNF) in
Parkinson's disease, using intermittent intraputamenal convection-enhanced delivery via a …