Importance of the pig as a human biomedical model
JK Lunney, A Van Goor, KE Walker… - Science translational …, 2021 - science.org
Pigs have substantial potential as biomedical models for studying human developmental
processes, congenital diseases, and pathogen response mechanisms in addition to utility as …
processes, congenital diseases, and pathogen response mechanisms in addition to utility as …
Viral vectors in gene therapy: Where do we stand in 2023?
K Lundstrom - Viruses, 2023 - mdpi.com
Viral vectors have been used for a broad spectrum of gene therapy for both acute and
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …
chronic diseases. In the context of cancer gene therapy, viral vectors expressing anti-tumor …
Natural history of infantile‐onset spinal muscular atrophy
Objective Infantile‐onset spinal muscular atrophy (SMA) is the most common genetic cause
of infant mortality, typically resulting in death preceding age 2. Clinical trials in this …
of infant mortality, typically resulting in death preceding age 2. Clinical trials in this …
Twenty-five years of spinal muscular atrophy research: from phenotype to genotype to therapy, and what comes next
Twenty-five years ago, the underlying genetic cause for one of the most common and
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …
devastating inherited diseases in humans, spinal muscular atrophy (SMA), was identified …
Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue
G Thomsen, AHM Burghes, C Hsieh, J Do, BTT Chu… - Nature medicine, 2021 - nature.com
Spinal muscular atrophy type 1 (SMA1) is a debilitating neurodegenerative disease resulting
from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec …
from survival motor neuron 1 gene (SMN1) deletion/mutation. Onasemnogene abeparvovec …
Antisense oligonucleotides: translation from mouse models to human neurodegenerative diseases
KM Schoch, TM Miller - Neuron, 2017 - cell.com
Multiple neurodegenerative diseases are characterized by single-protein dysfunction and
aggregation. Treatment strategies for these diseases have often targeted downstream …
aggregation. Treatment strategies for these diseases have often targeted downstream …
Gene therapy for spinal muscular atrophy: safety and early outcomes
MA Waldrop, C Karingada, MA Storey, B Powers… - …, 2020 - publications.aap.org
Gene Therapy for Spinal Muscular Atrophy: Safety and Early Outcomes | Pediatrics | American
Academy of Pediatrics Skip to Main Content Disclaimer » Advertising AAP logo Search Close …
Academy of Pediatrics Skip to Main Content Disclaimer » Advertising AAP logo Search Close …
Emerging therapies and challenges in spinal muscular atrophy
Spinal muscular atrophy (SMA) is a hereditary neurodegenerative disease with severity
ranging from progressive infantile paralysis and premature death (type I) to limited motor …
ranging from progressive infantile paralysis and premature death (type I) to limited motor …
Spinal muscular atrophy
SJ Kolb, JT Kissel - Neurologic clinics, 2015 - neurologic.theclinics.com
The natural history of SMA is complex and variable. For this reason, clinical subgroups have
been defined based upon best motor function attainment during development. Type 1 SMA …
been defined based upon best motor function attainment during development. Type 1 SMA …
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 …