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Modeling Glutaric Aciduria Type I in human neuroblastoma cells recapitulates neuronal damage that can be rescued by gene replacement.

• Authors : Mateu Bosch, Anna; Segur-Bailach, Eulalia; García Villoria, Judit

Subjects: Teràpia genètica; Malalties cerebrals; Proteïnes

• Source: Articles publicats en revistes (Medicina)

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Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy

• Authors : Le Guiner, Caroline; Xiao, Xiao; Larcher, Thibaut ...

Subjects: DMDmdx rat; Duchenne muscular dystrophy; age

• Source: EISSN: 2329-0501 ; Molecular Therapy - Methods and Clinical Development ; https://inserm.hal.science/inserm-04240276 ; Molecular Therapy -

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Intravitreal air tamponade after AAV2 subretinal injection modifies retinal EGFP distribution

• Authors : Ducloyer, Jean-Baptiste; Pichard, Virginie; Mevel, Mathieu ...

Subjects: air tamponade; eye surgery; fluid-air exchange

• Source: EISSN: 2329-0501 ; Molecular Therapy - Methods and Clinical Development ; https://hal.science/hal-04193954 ; Molecular Therapy - Methods

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Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

• Authors : Scala, Serena; Ferrua, Francesca; Basso-Ricci, Luca ...

Subjects: gene therapy

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Subretinal timrepigene emparvovec in adult men with choroideremia : a randomized phase 3 trial

• Authors : Maclaren, Robert E.; Fischer, M. Dominik; Gow, James A. ...

Subjects: Retinal gene-therapy; Natural-history; Expression

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Machine Learning and Metabolic Modelling Assisted Implementation of a Novel Process Analytical Technology in Cell and Gene Therapy Manufacturing

• Authors : Williams, Thomas; Kalinka, Kevin; Sanches, Rui

Subjects: Biochemical reaction networks; Biotechnology; Gene therapy

• Source: Scientific Reports , 13 (834) (2023)

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ANKS1A regulates LDL receptor-related protein 1 (LRP1)-mediated cerebrovascular clearance in brain endothelial cells

• Authors : Lee, Jiyeon; Lee, Haeryung; Lee, Hyein

Subjects: CEREBRAL AMYLOID ANGIOPATHY; ALZHEIMERS-DISEASE; A-BETA

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Clonal reconstruction from co-occurrence of vector integration sites accurately quantifies expanding clones in vivo

• Authors : Wagner, Sebastian; Baldow, Christoph; Calabria, Andrea

Subjects: Computational biology and bioinformatics, Gene therapy, Molecular medicine, hematopoiesis, clonal spectrum; Computergestützte Biologie und Bioinformatik, Gentherapie, Molekulare Medizin, Hämatopoese, klonales Spektrum; info:eu-repo/classification/ddc/500

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Systemic and local immune responses to intraocular AAV vector administration in non-human primates

• Authors : Ail, Divya; Ren, Duohao; Brazhnikova, Elena ...

Subjects: non-human primates; binding antibodies; neutralizing antibodies

• Source: EISSN: 2329-0501 ; Molecular Therapy - Methods and Clinical Development ; https://hal.sorbonne-universite.fr/hal-03602826 ; Molecular

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Reversal of RNA toxicity in myotonic dystrophy via a decoy RNA-binding protein with high affinity for expanded CUG repeats

• Authors : Arandel, Ludovic; Matloka, Magdalena; Klein, Arnaud, F ...

Subjects: Myotonic dystrophy; Gene therapy; MNBL

• Source: ISSN: 2157-846X ; Nature Biomedical Engineering ; https://hal.science/hal-03830811 ; Nature Biomedical Engineering, 2022, 6 (2), pp.207-220. ⟨10.1038/s41551-021-00838-2⟩.

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