Adenovirus-mediated gene delivery: potential applications for gene and cell-based therapies in the new era of personalized medicine

CS Lee, ES Bishop, R Zhang, X Yu, EM Farina, S Yan… - Genes & diseases, 2017 - Elsevier
With rapid advances in understanding molecular pathogenesis of human diseases in the era
of genome sciences and systems biology, it is anticipated that increasing numbers of …

Lipid nanoparticle-mediated drug delivery to the brain

P Khare, SX Edgecomb, CM Hamadani… - Advanced Drug Delivery …, 2023 - Elsevier
Lipid nanoparticles (LNPs) have revolutionized the field of drug delivery through their
applications in siRNA delivery to the liver (Onpattro) and their use in the Pfizer-BioNTech …

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 …

Blood–brain barrier shuttle peptides: an emerging paradigm for brain delivery

B Oller-Salvia, M Sánchez-Navarro, E Giralt… - Chemical Society …, 2016 - pubs.rsc.org
Brain delivery is one of the major challenges in drug development because of the high
number of patients suffering from neural diseases and the low efficiency of the treatments …

Pharmacotherapy for amyotrophic lateral sclerosis: a review of approved and upcoming agents

SA Johnson, T Fang, F De Marchi, D Neel… - Drugs, 2022 - Springer
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder
involving loss of upper and lower motor neurons, with most cases ending in death within 3–5 …

Viral vectors: a look back and ahead on gene transfer technology

L Vannucci, M Lai, F Chiuppesi, L Ceccherini-Nelli… - New …, 2013 - arpi.unipi.it
No matter what their origin, strain and family, viruses have evolved exquisite strategies to
reach and penetrate specific target cells where they hijack the cellular machinery to express …

Targeting dorsal root ganglia and primary sensory neurons for the treatment of chronic pain

T Berta, Y Qadri, PH Tan, RR Ji - Expert opinion on therapeutic …, 2017 - Taylor & Francis
Introduction: Currently the treatment of chronic pain is inadequate and compromised by
debilitating central nervous system side effects. Here we discuss new therapeutic strategies …

CRISPR-Cas9-mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model

FK Ekman, DS Ojala, MM Adil, PA Lopez… - … Therapy-Nucleic Acids, 2019 - cell.com
Huntington's disease (HD) is a currently incurable and, ultimately, fatal neurodegenerative
disorder caused by a CAG trinucleotide repeat expansion within exon 1 of the huntingtin …

Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9

J Saraiva, RJ Nobre, LP de Almeida - Journal of Controlled Release, 2016 - Elsevier
Several attempts have been made to discover the ideal vector for gene therapy in central
nervous system (CNS). Adeno-associated viruses (AAVs) are currently the preferred vehicle …

Gene therapy for Parkinson's disease, an update

TM Axelsen, DPD Woldbye - Journal of Parkinson's disease, 2018 - content.iospress.com
The current mainstay treatment of Parkinson's disease (PD) consists of dopamine
replacement therapy which, in addition to causing several side effects, does not delay …