Viral Vectors: The Road to Reducing Genotoxicity.
Rhiannon David,Ann T. Doherty +1 more
TLDR
There is not a 'one size fits all' approach to vector modification for reducing genotoxicity, and addressing these challenges will be a key step in the development of therapies such as CRISPR-Cas9 and delivery of future gene-editing technologies.About:
This article is published in Toxicological Sciences.The article was published on 2017-02-01 and is currently open access. It has received 93 citations till now. The article focuses on the topics: Insertional mutagenesis & Vector (molecular biology).read more
Citations
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Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins
Samagya Banskota,Aditya Raguram,Susie Suh,Samuel W. Du,Jessie Rose Davis,Elliot H. Choi,Xiao Wang,Sarah C. Nielsen,Gregory A. Newby,Peyton B. Randolph,Mark J. Osborn,Kiran Musunuru,Krzysztof Palczewski,David R. Liu +13 more
TL;DR: In this paper , the authors report the development and application of engineered DNA-free virus-like particles (eVLPs) that efficiently package and deliver base editor or Cas9 ribonucleoproteins.
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Polymer-stabilized Cas9 nanoparticles and modified repair templates increase genome editing efficiency.
David N. Nguyen,Theodore L. Roth,P. Jonathan Li,P. Jonathan Li,Peixin Amy Chen,Peixin Amy Chen,Ryan Apathy,Ryan Apathy,Murad R. Mamedov,Murad R. Mamedov,Linda T. Vo,Victoria Tobin,Victoria Tobin,Daniel B. Goodman,Daniel B. Goodman,Eric Shifrut,Eric Shifrut,Jeffrey A. Bluestone,Jennifer M. Puck,Francis C. Szoka,Alexander Marson +20 more
TL;DR: Two improvements are reported that increase the efficiency of CRISPR–Cas9-based genome editing in clinically relevant primary cell types and increases gene targeting efficiency even at reduced HDR template doses.
Journal ArticleDOI
Adipose-Derived Stem Cells: Current Applications and Future Directions in the Regeneration of Multiple Tissues.
TL;DR: An overview of the current status of ADSC preparation, including harvesting, isolation, and identification is provided and it is hoped that this review can provide comprehensive and standardized guidelines for the safe and effective application of ADSCs to achieve predictable and desired therapeutic effects.
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Design and Characterization of an "All-in-One" Lentiviral Vector System Combining Constitutive Anti-GD2 CAR Expression and Inducible Cytokines.
Katharina Zimmermann,Johannes Kuehle,Anna Christina Dragon,Melanie Galla,Christina Kloth,Loreen Sophie Rudek,I. Erol Sandalcioglu,Belal Neyazi,Thomas Moritz,Johann Meyer,Claudia Rossig,Bianca Altvater,Britta Eiz-Vesper,Michael A. Morgan,Hinrich Abken,Axel Schambach,Axel Schambach +16 more
TL;DR: It is shown that T cells containing the “all-in-one” vector system secrete the immunomodulatory cytokines interleukin (IL)12 or IL18 upon co-cultivation with primary human GD2+ tumor cells, resulting in enhanced effector cell properties and increased monocyte recruitment, highlighting the potential of the system to simplify application of TRUCK-modified T cells in solid tumor therapy.
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Advances in gene-based vaccine platforms to address the COVID-19 pandemic.
Deborah Pushparajah,Salma Jimenez,Shirley Wong,Hibah Alattas,Nafiseh Nafissi,Roderick A. Slavcev +5 more
TL;DR: In this article, a review of gene-based vaccine candidates proceeding through clinical trials, including their antigenic targets, delivery vehicles, and route of administration is discussed in guiding the design and development of effective vaccines against COVID-19 and future derivatives.
References
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HIV-1 Integration in the Human Genome Favors Active Genes and Local Hotspots
Astrid R. W Schroder,Paul Shinn,Huaming Chen,Charles C. Berry,Joseph R. Ecker,Frederic D. Bushman +5 more
TL;DR: Global analysis of cellular transcription indicated that active genes were preferential integration targets, particularly genes that were activated in cells after infection by HIV-1, and this data suggests how selective targeting promotes aggressive HIV replication.
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Transcription Start Regions in the Human Genome Are Favored Targets for MLV Integration
TL;DR: Map of retroviral integrations in the human genome showed that MLV preferred integration near the start of transcriptional units (either upstream or downstream) whereas HIV-1 preferred integration anywhere in the transcriptional unit but not upstream of a transcriptional start.
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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
Marina Cavazzana-Calvo,Emmanuel Payen,Olivier Negre,Gary P. Wang,Kathleen M. Hehir,Floriane Fusil,Julian D. Down,Maria Denaro,Troy Brady,Karen A. Westerman,Resy Cavallesco,Beatrix Gillet-Legrand,Laure Caccavelli,Laure Caccavelli,Riccardo Sgarra,Leila Maouche-Chretien,Françoise Bernaudin,Robert Girot,Ronald Dorazio,Geert Jan Mulder,Axel Polack,Arthur Bank,Jean Soulier,Jérôme Larghero,Nabil Kabbara,Bruno Dalle,Bernard Gourmel,Gérard Socié,Stany Chrétien,Stany Chrétien,Nathalie Cartier,Patrick Aubourg,Alain Fischer,Alain Fischer,Kenneth Cornetta,Frédéric Galactéros,Yves Beuzard,Eliane Gluckman,Frederick D. Bushman,Salima Hacein-Bey-Abina,Salima Hacein-Bey-Abina,Philippe Leboulch +41 more
TL;DR: It is shown that, 33 months after lentiviral β-globin gene transfer, an adult patient with severe βE/β0-thalassaemia dependent on monthly transfusions since early childhood has become transfusion independent for the past 21 months.
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Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy.
Alessandra Biffi,Eugenio Montini,Laura Lorioli,Martina Cesani,Francesca Fumagalli,Tiziana Plati,Cristina Baldoli,Sabata Martino,Andrea Calabria,Sabrina Canale,Fabrizio Benedicenti,Giuliana Vallanti,Luca Biasco,Simone Leo,Nabil Kabbara,Gianluigi Zanetti,William B. Rizzo,Nalini Mehta,Maria Pia Cicalese,Miriam Casiraghi,Jaap Jan Boelens,Ubaldo Del Carro,David J. Dow,Manfred Schmidt,Andrea Assanelli,Victor Neduva,Clelia Di Serio,Elia Stupka,Jason P. Gardner,Christof von Kalle,Claudio Bordignon,Claudio Bordignon,Fabio Ciceri,Attilio Rovelli,Maria Grazia Roncarolo,Alessandro Aiuti,Maria Sessa,Luigi Naldini +37 more
TL;DR: The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme.
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Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences.
Richard S. Mitchell,Brett Beitzel,Astrid R. W Schroder,Paul Shinn,Huaming Chen,Charles C. Berry,Joseph R. Ecker,Frederic D. Bushman +7 more
TL;DR: Each of the three retroviruses studied showed unique integration site preferences, suggesting that virus-specific binding of integration complexes to chromatin features likely guides site selection.