Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.

Thomsen G, Kaspar A, Ferraiuolo L, Chu B, Garcia VJ, Weiss R, Slaiwa T, Casanova-Vallve N, Cano R, Hurley E, Julazadeh H, Bautista H, Renteria A, Lee M, Rollins J, McAlonis-Downes M, Arnold WD, TreDenick T, Hsu R, Bayless LE, Kozial C, Forespring C, Molony M, Fraser M, Zhou J, Yao J, Bremner SN, Ward SR, Schenk S, Yoshikawa T, Moriyama A, Tsusaki H, Varma S, Kruger M, Stahl M, Kaspar B

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DOI: 10.1016/j.omta.2026.201707
Published: 2026 Mar 12
Container: Molecular therapy. Advances
Publisher: Not recorded
Open access: yes

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Cite this work

BibTeX

@article{allodium:10.1016/j.omta.2026.201707,
  title = {Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.},
  author = {Thomsen G and Kaspar A and Ferraiuolo L and Chu B and Garcia VJ and Weiss R and Slaiwa T and Casanova-Vallve N and Cano R and Hurley E and Julazadeh H and Bautista H and Renteria A and Lee M and Rollins J and McAlonis-Downes M and Arnold WD and TreDenick T and Hsu R and Bayless LE and Kozial C and Forespring C and Molony M and Fraser M and Zhou J and Yao J and Bremner SN and Ward SR and Schenk S and Yoshikawa T and Moriyama A and Tsusaki H and Varma S and Kruger M and Stahl M and Kaspar B},
  year = {2026},
  journal = {Molecular therapy. Advances},
  doi = {10.1016/j.omta.2026.201707},
  url = {https://doi.org/10.1016/j.omta.2026.201707}
}

RIS

TY  - JOUR
TI  - Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.
AU  - Thomsen G
AU  - Kaspar A
AU  - Ferraiuolo L
AU  - Chu B
AU  - Garcia VJ
AU  - Weiss R
AU  - Slaiwa T
AU  - Casanova-Vallve N
AU  - Cano R
AU  - Hurley E
AU  - Julazadeh H
AU  - Bautista H
AU  - Renteria A
AU  - Lee M
AU  - Rollins J
AU  - McAlonis-Downes M
AU  - Arnold WD
AU  - TreDenick T
AU  - Hsu R
AU  - Bayless LE
AU  - Kozial C
AU  - Forespring C
AU  - Molony M
AU  - Fraser M
AU  - Zhou J
AU  - Yao J
AU  - Bremner SN
AU  - Ward SR
AU  - Schenk S
AU  - Yoshikawa T
AU  - Moriyama A
AU  - Tsusaki H
AU  - Varma S
AU  - Kruger M
AU  - Stahl M
AU  - Kaspar B
PY  - 2026
JO  - Molecular therapy. Advances
DO  - 10.1016/j.omta.2026.201707
UR  - https://doi.org/10.1016/j.omta.2026.201707
ER  -

APA

G, T., A, K., L, F., B, C., VJ, G., R, W., T, S., N, C., R, C., E, H., H, J., H, B., A, R., M, L., J, R., M, M., WD, A., T, T., R, H., LE, B., C, K., C, F., M, M., M, F., J, Z., J, Y., SN, B., SR, W., S, S., T, Y., A, M., H, T., S, V., M, K., M, S., & B, K. (2026). Preclinical evaluation of INS1201 AAV9-micro-dystrophin via CSF administration as a potential therapy for Duchenne muscular dystrophy.. Molecular therapy. Advances. https://doi.org/10.1016/j.omta.2026.201707

Source records

pubmed · retrieved 2026-09-26T19:14:58.791Z