Zobrazeno 1 - 10
of 279
pro vyhledávání: '"Thierry, VandenDriessche"'
Autor:
Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K. Chuah, Dirk Grimm, Federico Mingozzi, Fatima Bosch
Publikováno v:
Molecular Metabolism, Vol 81, Iss , Pp 101899- (2024)
Objective: Pompe disease (PD) is caused by deficiency of the lysosomal enzyme acid α-glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy with progressive muscle weakness. In the Infantile-Onset PD (IOPD), death genera
Externí odkaz:
https://doaj.org/article/51f4889c5f3c445fbc764529166584d8
Autor:
Jonas Weinmann, Sabrina Weis, Josefine Sippel, Warut Tulalamba, Anca Remes, Jihad El Andari, Anne-Kathrin Herrmann, Quang H. Pham, Christopher Borowski, Susanne Hille, Tanja Schönberger, Norbert Frey, Martin Lenter, Thierry VandenDriessche, Oliver J. Müller, Marinee K. Chuah, Thorsten Lamla, Dirk Grimm
Publikováno v:
Nature Communications, Vol 11, Iss 1, Pp 1-12 (2020)
Adeno-associated virus is the basis of many gene therapies and gene transfer vectors. Here the authors report a pipeline to enable side-by-side comparison of pre-selected capsids in a high throughput manner.
Externí odkaz:
https://doaj.org/article/fcda7a44d2044540880d0f383706efd9
Autor:
Flora Peyvandi, David Lillicrap, Johnny Mahlangu, Claire McLintock, K. John Pasi, Steven W. Pipe, Wendy Scales, Alok Srivastava, Thierry VandenDriessche
Publikováno v:
Research and Practice in Thrombosis and Haemostasis, Vol 4, Iss 4, Pp 644-651 (2020)
Abstract Background Hemophilia gene therapy is a rapidly evolving therapeutic approach in which a number of programs are approaching clinical development completion. Objective The aim of this study was to evaluate knowledge and perceptions of a varie
Externí odkaz:
https://doaj.org/article/c1ff5ac1480648eb8dbeb547ea1ccccd
Autor:
Jaitip Tipanee, Mario Di Matteo, Warut Tulalamba, Ermira Samara-Kuko, Jiri Keirsse, Jo A. Van Ginderachter, Marinee Khim Chuah, Thierry VandenDriessche
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 19, Iss , Pp 1309-1329 (2020)
We established a semi-high-throughput in vivo screening platform using hyperactive piggyBac (hyPB) transposons (designated as PB-miR) to identify microRNAs (miRs) that inhibit hepatocellular carcinoma (HCC) development in vivo, following miR overexpr
Externí odkaz:
https://doaj.org/article/892c893b67c94af681db97ef39bae16c
Autor:
Kshitiz Singh, Denis Furling, Sumitava Dastidar, Marinee Chuah, Thierry VandenDriessche, Debanjana Majumdar, Jaitip Tipanee, Arnaud F. Klein
Publikováno v:
Molecular Therapy
Molecular Therapy, Cell Press, 2021, 29 (11), ⟨10.1016/j.ymthe.2021.08.004⟩
Molecular Therapy, 2021, 29 (11), ⟨10.1016/j.ymthe.2021.08.004⟩
Molecular Therapy, Cell Press, 2021, 29 (11), ⟨10.1016/j.ymthe.2021.08.004⟩
Molecular Therapy, 2021, 29 (11), ⟨10.1016/j.ymthe.2021.08.004⟩
CTGrepeat expansion (CTGexp) is associated with aberrant alternate splicing that contributes to cardiac dysfunction in myotonic dystrophy type 1 (DM1). Excision of thisCTGexprepeat using CRISPR-Cas resulted in the disappearance of punctate ribonuclea
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f587adb6edc9ad1dd712e72d4e19cdfe
https://doi.org/10.1016/j.ymthe.2021.08.004
https://doi.org/10.1016/j.ymthe.2021.08.004
Autor:
Shanthi Herath, HH Abdul-Razak, Susan Jarmin, Marinee Chuah, Linda Popplewell, Chiara Sidoli, Ngoc Lu-Nguyen, Alberto Malerba, George Dickson, Thierry VandenDriessche, Anita Le Heron
Publikováno v:
Human Gene Therapy. 32:1138-1146
Duchenne muscular dystrophy (DMD) is an X-linked recessive disease that affects 1:5,000 live male births and is characterized by muscle wasting. By the age of 13 years, affected individuals are often wheelchair bound and suffer from respiratory and c
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4e4112daffd9550d891767f9f1b9911c
https://doi.org/10.1089/hum.2020.325
https://doi.org/10.1089/hum.2020.325
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy. 30(12)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bd06917e35cbae3492e8f2bd7245d086
https://pubmed.ncbi.nlm.nih.gov/35821634
https://pubmed.ncbi.nlm.nih.gov/35821634
Autor:
Jaitip Tipanee, Ermira Samara-Kuko, Thierry Gevaert, Marinee K. Chuah, Thierry VandenDriessche
Allogeneic CD19-specific chimeric antigen receptor (CAR) T cells with inactivated donor T cell receptor (TCR) expression can be used as an "off-the-shelf" therapeutic modality for lymphoid malignancies, thus offering an attractive alternative to auto
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ac7c20b01c9712273deacef0e8b6d162
https://hdl.handle.net/20.500.14017/b85a3a4c-051f-455c-9710-29d2e688ae6d
https://hdl.handle.net/20.500.14017/b85a3a4c-051f-455c-9710-29d2e688ae6d
Publikováno v:
Molecular Therapy. 31:1-2
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::91de336c3b6a733458047f9fcd3e3d1c
https://doi.org/10.1016/j.ymthe.2022.12.001
https://doi.org/10.1016/j.ymthe.2022.12.001
Publikováno v:
The Lancet. Haematology. 9(10)
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::696358a062ae6d4d11c5ec31a4ee2c6c
https://pubmed.ncbi.nlm.nih.gov/36174639
https://pubmed.ncbi.nlm.nih.gov/36174639