Zobrazeno 1 - 10
of 638
pro vyhledávání: '"AAV9"'
Autor:
Monika Chauhan, Audrey L. Daugherty, Fatemeh (Ellie) Khadir, Ozgun F. Duzenli, Alexandra Hoffman, Jennifer A. Tinklenberg, Peter B. Kang, George Aslanidi, Christina A. Pacak
Publikováno v:
Journal of Translational Medicine, Vol 22, Iss 1, Pp 1-14 (2024)
Abstract Highly efficient adeno associated viruses (AAVs) targeting the central nervous system (CNS) are needed to deliver safe and effective therapies for inherited neurological disorders. The goal of this study was to compare the organ-specific tra
Externí odkaz:
https://doaj.org/article/cafeb29b8b2c4e6c854217899ac3adad
Autor:
Facundo Peralta, Ana Abril Vidal Escobedo, Juliette López Hanotte, Martino Avallone, Tomas Björklund, Paula Cecilia Reggiani, Joaquín Pardo
Publikováno v:
Neurobiology of Disease, Vol 200, Iss , Pp 106612- (2024)
Astrocytes play key roles in the brain. When astrocyte support fails, neurological disorders follow, resulting in disrupted synaptic communication, neuronal degeneration, and cell death. We posit that astrocytes overexpressing neurotrophic factors, s
Externí odkaz:
https://doaj.org/article/d5b3c2eefcde4459a98d04ac1c2d9a61
Autor:
Amit Chhabra, George Bashirians, Christos J. Petropoulos, Terri Wrin, Yuvika Paliwal, Peter V. Henstock, Suryanarayan Somanathan, Candida da Fonseca Pereira, Ian Winburn, John E.J. Rasko
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 3, Pp 101273- (2024)
Adeno-associated virus (AAV) vectors are promising gene therapy candidates, but pre-existing anti-AAV neutralizing antibodies (NAbs) pose a significant challenge to successful gene delivery. Knowledge of NAb seroprevalence remains limited and inconsi
Externí odkaz:
https://doaj.org/article/420602eea7e2485887542996c0356bcb
Autor:
Shin Jeon, Jaein Park, Shibi Likhite, Ji Hwan Moon, Dongjun Shin, Liwen Li, Kathrin C. Meyer, Jae W. Lee, Soo-Kyung Lee
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 3, Pp 101275- (2024)
Heterozygous mutations in the FOXG1 gene manifest as FOXG1 syndrome, a severe neurodevelopmental disorder characterized by structural brain anomalies, including agenesis of the corpus callosum, hippocampal reduction, and myelination delays. Despite t
Externí odkaz:
https://doaj.org/article/faddaeb42e0c4e1297b44f6d5392d3d0
Autor:
Stéphane J. Baudouin, April R. Giles, Nick Pearson, Severine Deforges, Chenxia He, Céline Boileau, Nicolas Partouche, Andreas Borta, Justine Gautron, Morgane Wartel, Irena Bočkaj, Didier Scavarda, Fabrice Bartolomei, Guillaume Penchet, Jérôme Aupy, Jennifer Sims, Jared Smith, Andrew Mercer, Olivier Danos, Christophe Mulle, Valérie Crépel, Richard Porter
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 4, Pp 101342- (2024)
Mesial temporal lobe epilepsy (mTLE) is the most prevalent type of epilepsy in adults. First and subsequent generations of anti-epileptic therapy regimens fail to decrease seizures in a large number of patients suffering from mTLE, leaving surgical a
Externí odkaz:
https://doaj.org/article/b7eefb3836e346488c8327be3214fc97
Publikováno v:
Open Medicine, Vol 18, Iss 1, Pp 1-11 (2023)
This study aims to investigate the impact of ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) on vascular calcification in rats. The rationale behind studying ENPP1’s role in vascular calcification lies in its potential to modulate calcif
Externí odkaz:
https://doaj.org/article/c0d0d14519f546979c71328bac7faa50
Autor:
Carola J. Maturana, Esteban A. Engel
Publikováno v:
Frontiers in Virology, Vol 4 (2024)
Significant progress has been made in enhancing recombinant adeno-associated virus (rAAV) for clinical investigation. Despite its versatility as a gene delivery platform, the inherent packaging constraint of 4.7 kb imposes restrictions on the range o
Externí odkaz:
https://doaj.org/article/cbdffdba455a4a9a9dff740aac963223
Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 38, Iss , Pp 101035- (2024)
Inherited deficiency of phosphomannomutase 2 (PMM2) (aka PMM2-CDG) is the most common congenital disorders of glycosylation (CDG) and has no cure. With debilitating morbidity and significant mortality, it is imperative to explore novel, safe, and eff
Externí odkaz:
https://doaj.org/article/d41aef350c394227aee71af1f7777a11
Autor:
Christina A. Pacak, Silveli Suzuki-Hatano, Fatemeh Khadir, Audrey L. Daugherty, Mughil Sriramvenugopal, Bennett J. Gosiker, Peter B. Kang, William Todd Cade
Publikováno v:
Journal of Translational Medicine, Vol 21, Iss 1, Pp 1-13 (2023)
Abstract Introduction The promising potential of adeno-associated virus (AAV) gene delivery strategies to treat genetic disorders continues to grow with an additional three AAV-based therapies recently approved by the Food and Drug Administration and
Externí odkaz:
https://doaj.org/article/f69833ae695641cf9607659cc8cf2067
Autor:
Alexia Kagiava, Christos Karaiskos, George Lapathitis, Amanda Heslegrave, Irene Sargiannidou, Henrik Zetterberg, Assumpció Bosch, Kleopas A. Kleopa
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 377-393 (2023)
X-linked Charcot-Marie-Tooth disease type 1 (CMT1X) is a demyelinating neuropathy resulting from loss-of-function mutations affecting the GJB1/connexin 32 (Cx32) gene. We previously showed functional and morphological improvement in Gjb1-null mice fo
Externí odkaz:
https://doaj.org/article/d4f32062b79d47ecacc281e8ce18674a