Zobrazeno 1 - 10
of 60
pro vyhledávání: '"Manuela Corti"'
B cell focused transient immune suppression protocol for efficient AAV readministration to the liver
Autor:
Jyoti Rana, Roland W. Herzog, Maite Muñoz-Melero, Kentaro Yamada, Sandeep R.P. Kumar, Anh K. Lam, David M. Markusic, Dongsheng Duan, Cox Terhorst, Barry J. Byrne, Manuela Corti, Moanaro Biswas
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101216- (2024)
Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders. Although the goal is to achieve lifelong correction with a single vector administration, the ability to redose would enable the extension of therapy in cases in
Externí odkaz:
https://doaj.org/article/dbfee83096ee41878357669ddf1b4522
Autor:
Shandra J. Trantham, Mackenzi A. Coker, Samantha Norman, Emma Crowley, Julie Berthy, Barry J. Byrne, Sub Subramony, XiangYang Lou, Manuela Corti
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 1, Pp 101179- (2024)
Gene therapy is a potential treatment for Friedreich ataxia, with multiple programs on the horizon. The purpose of this study was to collect opinions about gene therapy from individuals 14 years or older with Friedreich ataxia or parents/caregivers o
Externí odkaz:
https://doaj.org/article/f968620348454beeb01c65b48a304bd6
Autor:
Manuela Corti, Barry J. Byrne, Dominic J. Gessler, Grace Thompson, Samantha Norman, Jenna Lammers, Kirsten E. Coleman, Cristina Liberati, Melissa E. Elder, Maria L. Escolar, Ibrahim S. Tuna, Clementina Mesaros, Gary I. Kleiner, Deborah S. Barbouth, Heather L. Gray-Edwards, Nathalie Clement, Brian D. Cleaver, Guangping Gao
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 303-314 (2023)
Gene replacement therapy is a rational therapeutic strategy and clinical intervention for neurodegenerative disorders like Canavan disease, a leukodystrophy caused by biallelic mutations in the aspartoacylase (ASPA) gene. We aimed to investigate whet
Externí odkaz:
https://doaj.org/article/9c3eeeaf5a4542ada6da05e007811039
Publikováno v:
Viruses, Vol 15, Iss 12, p 2378 (2023)
Efficient and targeted delivery of a DNA payload is vital for developing safe gene therapy. Owing to the recent success of commercial oncolytic vector and multiple COVID-19 vaccines, adenovirus vectors are back in the spotlight. Adenovirus vectors ca
Externí odkaz:
https://doaj.org/article/5048d7ebbfa346a095adc3911ff3d32e
Autor:
Kathryn L. Bohnert, Grace Ditzenberger, Adam J. Bittel, Lisa de lasFuentes, Manuela Corti, Christina A. Pacak, Carolyn Taylor, Barry J. Byrne, Dominic N. Reeds, W. Todd Cade
Publikováno v:
JIMD Reports, Vol 62, Iss 1, Pp 74-84 (2021)
Abstract Background Muscle weakness and exercise intolerance contribute to reduced quality of life (QOL) in Barth syndrome (BTHS). Our group previously found that 12 weeks of resistance exercise training (RET) improved muscle strength, however, did n
Externí odkaz:
https://doaj.org/article/bda2cf0580534a32a6f3a6972555f1cf
Autor:
Nellie Georgiou-Karistianis, Louise A Corben, Kathrin Reetz, Isaac M Adanyeguh, Manuela Corti, Dinesh K Deelchand, Martin B Delatycki, Imis Dogan, Rebecca Evans, Jennifer Farmer, Marcondes C França, William Gaetz, Ian H Harding, Karen S Harris, Steven Hersch, Richard Joules, James J Joers, Michelle L Krishnan, Michelle Lax, Eric F Lock, David Lynch, Thomas Mareci, Sahan Muthuhetti Gamage, Massimo Pandolfo, Marina Papoutsi, Thiago J R Rezende, Timothy P L Roberts, Jens T Rosenberg, Sandro Romanzetti, Jörg B Schulz, Traci Schilling, Adam J Schwarz, Sub Subramony, Bert Yao, Stephen Zicha, Christophe Lenglet, Pierre-Gilles Henry
Publikováno v:
PLoS ONE, Vol 17, Iss 11, p e0269649 (2022)
IntroductionDrug development for neurodegenerative diseases such as Friedreich's ataxia (FRDA) is limited by a lack of validated, sensitive biomarkers of pharmacodynamic response in affected tissue and disease progression. Studies employing neuroimag
Externí odkaz:
https://doaj.org/article/13b6892396fd4fd9b5968cce8f1724fb
Autor:
Arnaud Gouelle, Samantha Norman, Bryanna Sharot, Stephanie Salabarria, Sub Subramony, Manuela Corti
Publikováno v:
Sensors, Vol 21, Iss 4, p 1144 (2021)
To date, it has been challenging for clinicians and researchers alike to use the multiple outcome measures available to create a meaningful clinical picture and perform effective longitudinal follow-up. It has been found that instrumented gait analys
Externí odkaz:
https://doaj.org/article/8617f92b936043899f2920577dc0e7a1
Publikováno v:
Brain Sciences, Vol 10, Iss 2, p 119 (2020)
Recently, adeno-associated virus (AAV)-mediated gene therapies have attracted clinical interest for treating neurodegenerative diseases including spinal muscular atrophy (SMA), Canavan disease (CD), Parkinson’s disease (PD), and Friedreich’s atax
Externí odkaz:
https://doaj.org/article/7da39926237247aba71e698b21450055
Autor:
Phillip A Doerfler, Sushrusha Nayak, Manuela Corti, Laurence Morel, Roland W Herzog, Barry J Byrne
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA) deficiency). Unfortunately, the introduction of GAA and viral vectors encoding the enzyme can lead
Externí odkaz:
https://doaj.org/article/d33f7b4f31044bd883a85f17c966f1d6
Publikováno v:
Physiology. 38
Pompe disease is a neuromuscular disorder resulting from mutations in the gene for acid a-glucosidase (GAA) – an enzyme necessary to degrade lysosomal glycogen. Early-onset disease occurs in the absence of functional GAA, which leads to cardiorespi