Expert Opinion on Biological Therapy, Volume 22, Issue 9 (2022)

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Expert Opinion on Biological Therapy, Volume 22, Issue 9 (2022)

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Gene therapy

Editorial

Editorial
Addressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’?
Takashi Kei Kishimoto & Richard Jude Samulski
Pages: 1067-1071
Published online: 03 Apr 2022
First Page PreviewforAddressing high dose AAV toxicity – ‘one and done’ or ‘slower and lower’? | Full Text | References | PDF (887.6 KB) | EPUB
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Editorial
Enhancing gene therapy regulatory interactions
Peter Marks
Pages: 1073-1074
Published online: 03 Apr 2022
CitationforEnhancing gene therapy regulatory interactions | Full Text | References | PDF (117.1 KB) | EPUB
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Drug Evaluation

Article
Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
Hugh J. McMillan, Crystal M. Proud, Michelle A. Farrar, Ian E. Alexander, Francesco Muntoni & Laurent Servais
Pages: 1075-1090
Published online: 02 May 2022
AbstractforOnasemnogene abeparvovec for the treatment of spinal muscular atrophy | Full Text | References | PDF (469.3 KB) | EPUB
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Open Access

Perspective

Article
Challenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies – a spotlight on hemophilia
Oscar G Segurado, Ruhong Jiang & Steven W Pipe
Pages: 1091-1098
Published online: 21 Jun 2022
AbstractforChallenges and opportunities when transitioning from in vivo gene replacement to in vivo CRISPR/Cas9 therapies – a spotlight on hemophilia | Full Text | References | PDF (1.4 MB) | EPUB
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Review

Article
Hemophilia A gene therapy: current and next-generation approaches
Steven W. Pipe, Gil Gonen-Yaacovi & Oscar G. Segurado
Pages: 1099-1115
Published online: 06 Jan 2022
AbstractforHemophilia A gene therapy: current and next-generation approaches | Full Text | References | PDF (1.5 MB) | EPUB
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Open Access
Review
What’s new and what’s next for gene therapy in Pompe disease?
Angela L. Roger, Ronit Sethi, Meredith L. Huston, Evelyn Scarrow, Joy Bao-Dai, Elias Lai, Debolina D. Biswas, Léa El Haddad, Laura M. Strickland, Priya S. Kishnani & Mai K. ElMallah
Pages: 1117-1135
Published online: 27 Apr 2022
AbstractforWhat’s new and what’s next for gene therapy in Pompe disease? | Full Text | References | PDF (616.3 KB) | EPUB
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Review
Current developments in gene therapy for epidermolysis bullosa
Thomas Kocher, Igor Petkovic, Johannes Bischof & Ulrich Koller
Pages: 1137-1150
Published online: 14 Mar 2022
AbstractforCurrent developments in gene therapy for epidermolysis bullosa | Full Text | References | PDF (2.9 MB) | EPUB
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Review
Treatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy
Ashish O Gupta, Gerald Raymond, Elizabeth I Pierpont, Stephan Kemp, R Scott McIvor, Arpana Rayannavar, Bradley Miller, Troy C Lund & Paul J Orchard
Pages: 1151-1162
Published online: 19 Sep 2022
AbstractforTreatment of cerebral adrenoleukodystrophy: allogeneic transplantation and lentiviral gene therapy | Full Text | References | PDF (2.5 MB) | EPUB
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Review
Circumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders
Lara Marrone, Paolo M. Marchi & Mimoun Azzouz
Pages: 1163-1176
Published online: 06 Jan 2022
AbstractforCircumventing the packaging limit of AAV-mediated gene replacement therapy for neurological disorders | Full Text | References | PDF (6.1 MB) | EPUB
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Review
Development and Clinical Translation Considerations for the Next Wave of Gene Modified Hematopoietic Stem and Progenitor Cells Therapies
Matthew Li, Brent Morse & Sadik Kassim
Pages: 1177-1191
Published online: 22 Jul 2022
AbstractforDevelopment and Clinical Translation Considerations for the Next Wave of Gene Modified Hematopoietic Stem and Progenitor Cells Therapies | Full Text | References | PDF (1.6 MB) | EPUB
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Review
Advancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques
Kyle D Kovacs, Thomas A Ciulla & Szilárd Kiss
Pages: 1193-1208
Published online: 19 Sep 2022
AbstractforAdvancements in ocular gene therapy delivery: vectors and subretinal, intravitreal, and suprachoroidal techniques | Full Text | References | PDF (999.9 KB) | EPUB
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Review
Recent advances in delivering RNA-based therapeutics to mitochondria
Yuma Yamada, Sen Ishizuka, Manae Arai, Minako Maruyama & Hideyoshi Harashima
Pages: 1209-1219
Published online: 11 May 2022
AbstractforRecent advances in delivering RNA-based therapeutics to mitochondria | Full Text | References | PDF (3.6 MB) | EPUB
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