Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

Irene Faravelli; Paola Rinchetti; Monica Tambalo; Illia Simutin; Lisa Mapelli; Sara Mancinelli; Matteo Miotto; Mafalda Rizzuti; Andrea D’Angelo; Chiara Cordiglieri; Giulia Forotti; Clelia Peano; Paolo Kunderfranco; Luca Calandriello; Giacomo P. Comi; Elvezia Paraboschi; Eleonora Pali; Francesca Beatrice; Egidio D’Angelo; Serge Przedborski; Monica Nizzardo; Simona Lodato; Stefania Corti

doi:10.1038/s41467-025-67725-1

Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

I Irene Faravelli P Paola Rinchetti M Monica Tambalo I Illia Simutin L Lisa Mapelli S Sara Mancinelli M Matteo Miotto M Mafalda Rizzuti A Andrea D’Angelo C Chiara Cordiglieri G Giulia Forotti C Clelia Peano P Paolo Kunderfranco L Luca Calandriello G Giacomo P. Comi E Elvezia Paraboschi E Eleonora Pali F Francesca Beatrice E Egidio D’Angelo S Serge Przedborski M Monica Nizzardo S Simona Lodato S Stefania Corti

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Article Details

Journal Nature Communications

Volume / Issue Vol. 17, Issue 1

Published December 21, 2025

DOI 10.1038/s41467-025-67725-1

ISSN 2041-1723

Publisher Nature Portfolio

Journal Info

Nature Communications

Nature Portfolio

ISSN: 2041-1723 Open Access Life Sciences

Authors (23)

I

Irene Faravelli

P

Paola Rinchetti

M

Monica Tambalo

I

Illia Simutin

L

Lisa Mapelli

S

Sara Mancinelli

M

Matteo Miotto

M

Mafalda Rizzuti

A

Andrea D’Angelo

C

Chiara Cordiglieri

G

Giulia Forotti

C

Clelia Peano

P

Paolo Kunderfranco

L

Luca Calandriello

G

Giacomo P. Comi

E

Elvezia Paraboschi

E

Eleonora Pali

F

Francesca Beatrice

E

Egidio D’Angelo

S

Serge Przedborski

M

Monica Nizzardo

S

Simona Lodato

S

Stefania Corti

Targeted antisense oligonucleotide treatment rescues developmental alterations in spinal muscular atrophy organoids

Article Details

Journal Info

Authors (23)

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