PGN-EDO51: Preclinical Data Supporting the Development of an Enhanced Delivery Oligonucleotide (EDO) for the Treatment of Duchenne Muscular Dystrophy (DMD)

被引：2

作者：

Holland, Ashling ^{[1
]}

Godfrey, Caroline ^{[1
]}

Lonkar, Pallavi ^{[1
]}

Svenstrup, Niels ^{[1
]}

Larkindale, Jane ^{[1
]}

McArthur, James ^{[1
]}

Bracegirdle, Sonia ^{[1
]}

Goyal, Jaya ^{[1
]}

机构：

[1] PepGen Inc, Cambridge, MA USA

来源：

NEUROLOGY | 2023年 / 100卷 / 17期

关键词：

D O I：

10.1212/WNL.0000000000203724

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

P3-8.002

引用

页数：3

共 26 条

[21] Preclinical Studies on Intestinal Administration of Antisense Oligonucleotides as a Model for Oral Delivery for Treatment of Duchenne Muscular Dystrophy
van Putten, Maaike
Young, Courtney
van den Berg, Sjoerd
Pronk, Amanda
Hulsker, Margriet
Karnaoukh, Tatyana G.
Vermue, Rick
van Dijk, Ko Willems
de Kimpe, Sjef
Aartsma-Rus, Annemieke
MOLECULAR THERAPY-NUCLEIC ACIDS, 2014, 3 : e211
[22] Three novel enhanced delivery Oligonucleotide candidates for Duchenne muscular dystrophy mediate high levels of exon 53, 45, and 44 skipping
Holland, A.
Lonkar, P.
Sweeney, C.
Zhang, H.
Svenstrup, N.
Gibbons, C.
Xu, L.
Foy, J.
Goyal, J.
NEUROMUSCULAR DISORDERS, 2023, 33 : S103 - S104
[23] Survival in eteplirsen-treated vs Duchenne Muscular Dystrophy (DMD) natural history patients: an indirect treatment comparison using real-world data
Iff, J.
Done, N.
Tuttle, E.
Zhong, Y.
Wei, F.
Darras, B.
McDonald, C.
Mercuri, E.
Muntoni, F.
NEUROMUSCULAR DISORDERS, 2022, 32
[24] Development of a validated Western blot method for quantification of human dystrophin protein used in Phase II and III clinical trials of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD)
Schnell, F. J.
Donoghue, C.
Dworzak, J.
Charleston, J. S.
Frank, D. E.
Wilton, S.
Lewis, S.
Mendell, J. R.
Rodino-Klapac, L. R.
Sahenk, Z.
NEUROLOGY, 2017, 88
[25] Pas de Deux - A Single Inducible High-Capacity Adenoviral Vector for Delivery of a Functional TALEN Pair Directed Against Human Dystrophin for the Treatment of Duchenne Muscular Dystrophy (DMD)
Schulz, Eric
Leitner, Theo
Erdmann-Brendel, Juliane
Bergmann, Thorsten
Gebbing, Maren
Gersbach, Charles A.
Ehrhardt, Anja
MOLECULAR THERAPY, 2015, 23 : S43 - S43
[26] Preclinical data for a novel Toll-like receptor antagonist in mdx mice support its clinical development as a potential treatment for disease-related muscle inflammation in Duchenne muscular dystrophy
Boehler, J.
Nagaraju, K.
Hurtt, M.
Agrawal, S.
NEUROMUSCULAR DISORDERS, 2015, 25 : S292 - S292

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