AAV-vectored base editor trans-splicing delivers dystrophin repair

AAV-vectored base editor trans-splicing delivers dystrophin repair

Bibliographic Details
Main Authors:	Zhen Li, Manuel A.F.V. Gonçalves
Format:	Article
Language:	English
Published:	Elsevier 2023-06-01
Series:	Molecular Therapy: Nucleic Acids
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253123001300

Similar Items

mRNA trans-splicing dual AAV vectors for (epi)genome editing and gene therapy
by: Lisa Maria Riedmayr, et al.
Published: (2023-10-01)

Extending AAV Packaging Cargo through Dual Co-Transduction: Efficient Protein Trans-Splicing at Low Vector Doses
by: Mariana V. Ferreira, et al.
Published: (2023-06-01)

Triple trans-splicing adeno-associated virus vectors capable of transferring the coding sequence for full-length dystrophin protein into dystrophic mice.
by: Koo, T, et al.
Published: (2014)

Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease
by: Chady H. Hakim, et al.
Published: (2020-09-01)

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors
by: Makoto Horiuchi, et al.
Published: (2022-12-01)

Evaluating the transduction efficiency of systemically delivered AAV vectors in the rat nervous system
by: Olivia J. Yang, et al.
Published: (2023-01-01)

Intravenous immunoglobulin prevents peripheral liver transduction of intrathecally delivered AAV vectors
by: Makoto Horiuchi, et al.
Published: (2022-12-01)

T Cell-Mediated Immune Responses to AAV and AAV Vectors
by: Hildegund C. J. Ertl
Published: (2021-04-01)

Expression and function of four AAV-based constructs for dystrophin restoration in the mdx mouse model of Duchenne muscular dystrophy
by: Rachael A. Potter, et al.
Published: (2023-09-01)

Evaluation of an AAV9-mini-dystrophin gene therapy candidate in a rat model of Duchenne muscular dystrophy
by: Caroline Le Guiner, et al.
Published: (2023-09-01)

Considerations for a Successful RNA Trans-splicing Repair of Genetic Disorders
by: Ulrich Koller, et al.
Published: (2014-01-01)

Alternative splicing of dystrobrevin regulates the stoichiometry of syntrophin binding to the dystrophin protein complex.
by: Newey, S, et al.
Published: (2000)

Impact of volume and expression time in an AAV-delivered channelrhodopsin
by: Sanaz Ansarifar, et al.
Published: (2023-11-01)

Exon exchange approach to repair Duchenne dystrophin transcripts.
by: Stéphanie Lorain, et al.
Published: (2010-01-01)

Innate immune responses to AAV vectors
by: Geoffrey L Rogers, et al.
Published: (2011-09-01)

Targeting the somatosensory system with AAV9 and AAV2retro viral vectors.
by: Alexander G J Skorput, et al.
Published: (2022-01-01)

Targeting the somatosensory system with AAV9 and AAV2retro viral vectors
by: Alexander G. J. Skorput, et al.
Published: (2022-01-01)

RNA-Seq Analysis of Trans-Differentiated ARPE-19 Cells Transduced by AAV9-AIPL1 Vectors
by: Alima Galieva, et al.
Published: (2023-12-01)

Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching
by: Kole Ryszard, et al.
Published: (2011-10-01)

Electroporation Enhanced Effect of Dystrophin Splice Switching PNA Oligomers in Normal and Dystrophic Muscle
by: Camilla Brolin, et al.
Published: (2015-01-01)

Adenoviral Vectors Meet Gene Editing: A Rising Partnership for the Genomic Engineering of Human Stem Cells and Their Progeny
by: Francesca Tasca, et al.
Published: (2020-04-01)

AAV vector-mediated in vivo reprogramming into pluripotency
by: Elena Senís, et al.
Published: (2018-07-01)

Thorough molecular configuration analysis of noncanonical AAV genomes in AAV vector preparations
by: Junping Zhang, et al.
Published: (2024-03-01)

RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes
by: Megha Subramanian, et al.
Published: (2023-04-01)

Dystrophin and dystrophin-associated protein in muscles and nerves from monkey
by: M Royuela, et al.
Published: (2009-06-01)

AAV Vectored Immunoprophylaxis for Filovirus Infections
by: Amira D. Rghei, et al.
Published: (2020-11-01)

AAV gene therapy vectors in the TMJ
by: Sabine M. Brouxhon, et al.
Published: (2022-12-01)

Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors.
by: Handa, A, et al.
Published: (2000)

Adeno-associated virus (AAV)-3-based vectors transduce haematopoietic cells not susceptible to transduction with AAV-2-based vectors
by: Handa, A, et al.
Published: (2000)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping.
by: Goyenvalle, A, et al.
Published: (2012)

Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping
by: Goyenvalle, A, et al.
Published: (2012)

Trans-ocular Electric Current In Vivo Enhances AAV-Mediated Retinal Gene Transduction after Intravitreal Vector Administration
by: Hongman Song, et al.
Published: (2019-06-01)

Development of a single vector system that enhances trans-splicing of SMN2 transcripts.
by: Tristan H Coady, et al.
Published: (2008-01-01)

Successful Transduction with AAV Vectors after Selective Depletion of Anti-AAV Antibodies by Immunoadsorption
by: Alejandro Orlowski, et al.
Published: (2020-03-01)

The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner
by: Adriana Gonzalez-Sandoval, et al.
Published: (2023-04-01)

Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes
by: Valeria V. Kolesnik, et al.
Published: (2024-03-01)

Function and genetics of dystrophin and dystrophin-related proteins in muscle.
by: Blake, D, et al.
Published: (2002)

Lentiviral vectors can be used for full-length dystrophin gene therapy
by: John R. Counsell, et al.
Published: (2017-03-01)

AAV VECTORS VACCINES AGAINST INFECTIOUS DISEASES
by: Karen eNieto, et al.
Published: (2014-01-01)

AAV- based vector improvements unrelated to capsid protein modification
by: Ekaterina M. Shitik, et al.
Published: (2023-02-01)