Details

Autor(en) / Beteiligte

• Bharucha-Goebel, Diana X.
• Todd, Joshua J.
• Saade, Dimah
• Norato, Gina
• Jain, Minal
• Lehky, Tanya
• Bailey, Rachel M.
• Chichester, Jessica A.
• Calcedo, Roberto
• Armao, Diane
• Foley, A. Reghan
• Mohassel, Payam
• Tesfaye, Eshetu
• Carlin, Bradley P.
• Seremula, Beth
• Waite, Melissa
• Zein, Wadih M.
• Huryn, Laryssa A.
• Crawford, Thomas O.
• Sumner, Charlotte J.
• Hoke, Ahmet
• Heiss, John D.
• Charnas, Lawrence
• Hooper, Jody E.
• Bouldin, Thomas W.
• Kang, Elizabeth M.
• Rybin, Denis
• Gray, Steven J.
• Bönnemann, Carsten G.

Titel

Intrathecal Gene Therapy for Giant Axonal Neuropathy

Ist Teil von

• The New England journal of medicine, 2024-03, Vol.390 (12), p.1092-1104

Ort / Verlag

United States: Massachusetts Medical Society

Erscheinungsjahr

2024

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• In a phase 1 study involving children with giant axonal neuropathy, intrathecal administration of an adeno-associated virus containing a GAN transgene resulted in some improvement in motor function scores.