Investigating the Impact of Delivery Routes for Exon Skipping Therapies in the CNS of DMD Mouse Models

Nucleic acid-based therapies have demonstrated great potential for the treatment of monogenetic diseases, including neurologic disorders. To date, regulatory approval has been received for a dozen antisense oligonucleotides (ASOs); however, these chemistries cannot readily cross the blood–brain barr...

وصف كامل

التفاصيل البيبلوغرافية

المؤلفون الرئيسيون:	Amel Saoudi, Claire Fergus, Talia Gileadi, Federica Montanaro, Jennifer E. Morgan, Vincent P. Kelly, Thomas Tensorer, Luis Garcia, Cyrille Vaillend, Francesco Muntoni, Aurélie Goyenvalle
التنسيق:	Artigo
اللغة:	Inglês
منشور في:	MDPI AG 2023-03-01
سلاسل:	Cells
الموضوعات:	ASO-based therapy central nervous system intracerebroventricular injection intrathecal injection antisense oligonucleotides exon-skipping
الوصول للمادة أونلاين:	https://www.mdpi.com/2073-4409/12/6/908
الوسوم:	إضافة وسم لا توجد وسوم, كن أول من يضع وسما على هذه التسجيلة!