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 …
of genome sciences and systems biology, it is anticipated that increasing numbers of …
Lipid nanoparticle-mediated drug delivery to the brain
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 …
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
Parkinson disease (PD) treatment options have conventionally focused on dopamine
replacement and provision of symptomatic relief. Current treatments cause undesirable …
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 …
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
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 …
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 …
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
Introduction: Currently the treatment of chronic pain is inadequate and compromised by
debilitating central nervous system side effects. Here we discuss new therapeutic strategies …
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 …
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 …
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 …
replacement therapy which, in addition to causing several side effects, does not delay …