Spinal muscular atrophy approval boosts antisense drugs
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Several approaches to circumvent the blood-brain barrier have been utilized clinically: intracerebroventricular Cerliponase for Batten's disease, intrathecal Spinraza for infantile spinal muscular atrophy, intrathecal Ziconotide for chronic pain, and intranasal insulin, leptin, and oxytocin.
“These include intracerebroventricular administration of Cerliponase for Batten's disease (395), intrathecal administration of antisense oligo Spinraza targeting the survival motor neuron 1 gene (SMN1) for infantile spinal muscular atrophy (150), intrathecal Ziconited peptide for chronic pain (398), and intranasal administration of insulin, leptin, and oxytocin (103).”
From Blood-Brain Barrier: From Physiology to Disease and Back (2019) · cites this paper as [150]
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Blood-Brain Barrier: From Physiology to Disease and Back
Sweeney, M. D., Zhao, Z., Montagne, A., Nelson, A. R., & Zlokovic, B. V. (2019) · Physiological Reviews · doi:10.1152/physrev.00050.2017 · Cited by 2,294 · see citing works
Advances in the delivery of RNA therapeutics: from concept to clinical reality
Kaczmarek, J. C., Kowalski, P. S., & Anderson, D. G. (2017) · Genome Medicine · doi:10.1186/s13073-017-0450-0 · Cited by 656 · see citing works
- Authors
- Dolgin, E.
- Corresponding author
- Dolgin, Elie
- Venue
- Nature Biotechnology
- Published
- 2017-02
- Volume
- 35
- Issue
- 2
- Pages
- 99-100
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- OpenAlex
- W2587419486
- Cited by
- 23
- References
- 0
- Citations by year
- 2025: 2 · 2022: 2 · 2021: 1 · 2020: 2 · 2019: 3 · 2018: 5 · 2017: 8
- Type
- other
- Language
- en
- Open access
- closed
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Biotechnology (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Health Sciences › Medicine › Genetics › Neurogenetic and Muscular Disorders Research
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Health Sciences › Medicine › Neurology › Amyotrophic Lateral Sclerosis Research
- Keywords
- spinal muscular atrophy, antisense oligonucleotides, drug approval, SMN2, SMN protein, alternative splicing, RNA therapeutics, myotonic dystrophy type 1, gene therapy
- MeSH terms
- Drug Industry, Humans, Muscular Atrophy, Spinal, United States, United States Food and Drug Administration, Oligonucleotides, Antisense, Drug Approval
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