Literature DB >> 30006718

Intrathecal Delivery of Viral Vectors for Gene Therapy.

Alexia Kagiava1, Kleopas A Kleopa2,3.   

Abstract

Gene delivery to the peripheral nervous system for therapeutic applications remains technically challenging but could eventually have a significant impact on the development of innovative treatments not only for inherited but also for acquired peripheral neuropathies. Here we describe the method for lumbar intrathecal injection of viral vectors in experimental mice. This gene delivery route provides widespread and stable over time Schwann cell-targeted or ubiquitous gene expression in the peripheral nervous system.

Entities:  

Keywords:  Gene delivery; Gene therapy; Intrathecal injection; Lentiviral vectors; Peripheral nerves; Schwann cells; Spinal roots

Mesh：

Year:  2018        PMID： 30006718     DOI： 10.1007/978-1-4939-7862-5_22

Source DB:  PubMed          Journal:  Methods Mol Biol        ISSN： 1064-3745


  4 in total

1.  Krabbe disease successfully treated via monotherapy of intrathecal gene therapy.

Authors:  Allison M Bradbury; Jessica H Bagel; Duc Nguyen; Erik A Lykken; Jill Pesayco Salvador; Xuntian Jiang; Gary P Swain; Charles A Assenmacher; Ian J Hendricks; Keiko Miyadera; Rebecka S Hess; Arielle Ostrager; Patricia ODonnell; Mark S Sands; Daniel S Ory; G Diane Shelton; Ernesto R Bongarzone; Steven J Gray; Charles H Vite
Journal:  J Clin Invest       Date:  2020-09-01       Impact factor: 14.808

2.  Efficacy of AAV serotypes to target Schwann cells after intrathecal and intravenous delivery.

Authors:  A Kagiava; J Richter; C Tryfonos; M Leal-Julià; I Sargiannidou; C Christodoulou; A Bosch; K A Kleopa
Journal:  Sci Rep       Date:  2021-12-02       Impact factor: 4.379

3.  Targeted PMP22 TATA-box editing by CRISPR/Cas9 reduces demyelinating neuropathy of Charcot-Marie-Tooth disease type 1A in mice.

Authors:  Ji-Su Lee; Jae Y Lee; Dong W Song; Hee S Bae; Hyun M Doo; Ho S Yu; Kyu J Lee; Hee K Kim; Hyun Hwang; Geon Kwak; Daesik Kim; Seokjoong Kim; Young B Hong; Jung M Lee; Byung-Ok Choi
Journal:  Nucleic Acids Res       Date:  2020-01-10       Impact factor: 16.971

4.  AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy.

Authors:  Alexia Kagiava; Christos Karaiskos; Jan Richter; Christina Tryfonos; Matthew J Jennings; Amanda J Heslegrave; Irene Sargiannidou; Marina Stavrou; Henrik Zetterberg; Mary M Reilly; Christina Christodoulou; Rita Horvath; Kleopas A Kleopa
Journal:  Gene Ther       Date:  2021-03-10       Impact factor: 5.250
  4 in total

北京卡尤迪生物科技股份有限公司 © 2022-2023.