Primary Cells Engineering and Molecular Delivery for Cellular and Molecular Therapies In Vivo and Ex Vivo

TOPICAL COLLECTIONS

Guest Editor

Valentina Romeo

Roche Pharma Research and Early Development, F.Hoffmann-La Roche Ltd, Grenzacherstrasse 124 4070 Basel, Switzerland

I received a Bachelor’s degree in Biological Sciences in 2008 and an MS in Molecular Biology in 2010 at the University of Milano-Bicocca, Italy. During that period, my research focused on mechanisms of gene expression and pre-mRNA splicing as they relate to neurodegenerative diseases. I obtained my PhD degree in Molecular Biology and Biochemistry in 2015 from the University of Bern-Switzerland, for studies related to cell cycle regulation and 3’end processing of mRNAs. In 2015 I moved to San Francisco-California and joined Genentech Inc. as a postdoctoral fellow in the Department of Research Pathology where I dedicated my research to (i) investigate the effect of combination therapy on human primary NK cells activation and signaling pathways and (ii) develop a method for in situ ISH-ICC on paucicellular specimens for biomarker detection. Between 2018 and 2020 I undertook a second postdoc in the Departments of Cancer Immunology and Molecular Biology at Genentech Inc. where I contributed to the development of mRNA-based cancer vaccines for personalized therapy and the understanding of their mechanisms of action. Currently, I am a Senior Scientist in Roche, Lead Discovery.

Collection Overview

Recent major advances in molecular and cell engineering capacities paved the way for the development of innovative therapeutic modalities and the rise of personalized medicine to correct genetic and acquired diseases.

Among those groundbreaking therapies, some rely on in vitro manipulation of isolated primary cells that are subsequently re-infused. Other therapies aim to target specific cell types in vivo; either to deliver curative payloads (e.g., gene therapy, vaccines) or to trigger direct conversion of existing cells into a different cell type in situ.

This collection aims to provide the reader with methods for ex vivo and in vivo primary cell engineering. These include methodologies to (i) isolate, maintain and engineer primary cells via viral and non-viral approaches, (ii) induce direct conversion of cells in vitro and in vivo and (iii) deliver payloads in vitro and in vivo.

Articles

8:20

Genome Engineering of Primary Human B Cells Using CRISPR/Cas9

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Cited by 13

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2020

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Kanut Laoharawee^1,2,3, Matthew J. Johnson^1,2,3, Walker S. Lahr^1,2,3, Joseph J. Peterson^1,2,3, Beau R. Webber^1,2,3, Branden S. Moriarity^1,2,3

¹Department of Pediatrics, University of Minnesota, ²Center for Genomic Engineering, University of Minnesota, ³Masonic Cancer Center, University of Minnesota

10:28

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

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Cited by 3

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2021

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Camila F. Almeida¹, Emma C. Frair¹, Nianyuan Huang¹, Reid Neinast², Kim L. McBride^2,3,4,6, Robert B. Weiss⁵, Kevin M. Flanigan^1,6, Nicolas Wein^1,6

¹Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, ²Center for Cardiovascular Research, The Research Institute at Nationwide Children’s Hospital, ³The Heart Center, Nationwide Children’s Hospital, ⁴Division of Genetic and Genomic Medicine, Nationwide Children’s Hospital, ⁵Department of Human Genetics, The University of Utah School of Medicine, ⁶Department of Pediatrics, The Ohio State University

11:42

In vitro Modeling for Neurological Diseases using Direct Conversion from Fibroblasts to Neuronal Progenitor Cells and Differentiation into Astrocytes

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Cited by 9

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2021

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Cassandra N. Dennys¹, Julieth A. Sierra-Delgado¹, Shrestha Sinha Ray¹, Annalisa M. Hartlaub¹, Florence S. Roussel¹, Yacidzohara Rodriguez¹, Kathrin Meyer^1,2

¹Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital, ²College of Medicine, The Ohio State University

9:46

Isolation, Culture, and Genetic Engineering of Mammalian Primary Pigment Epithelial Cells for Non-Viral Gene Therapy

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Cited by 2

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2021

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Thais Bascuas*^1,2, Martina Kropp*^1,2, Nina Harmening^1,2, Brittany M. Wong¹, Sandra Johnen³, Zsuzsanna Izsvák⁴, Gabriele Thumann^1,2

¹Experimental Ophthalmology, University of Geneva, ²Department of Ophthalmology, University Hospitals of Geneva, ³Department of Ophthalmology, University Hospital RWTH Aachen, ⁴Max Delbrück Center for Molecular Medicine

Abstracts

Isolation, cultivation and viral vector-based gene delivery to human limbal/progenitor stem cells

Matthew Hirsch^*1

¹University of North Carolina

In vitro separation and culturing of primary chondrocytes for cartilage biology

Qingxin Song¹,

Yangwei Xu¹,

Jian Sun²,

Yitong Zhao^*3,

Wenfang Wang^*1

¹Key laboratory of Shaanxi Province for Craniofacial Precision Medicine Research；Department of Orthodontics, College of Stomatology, Xi'an Jiaotong University 98 Xiwu Road, Xi’an, 710004, People’s Republic of China,

²Institute of Endemic Diseases, School of Public Health , Xi’an Jiaotong University Health Science Center, West Yanta Street No.76, Xi’an, Shaanxi 710061, PR China,

³Department of Biochemistry and Molecular Biology, School of Basic Medical Sciences, Xi’an Jiaotong University Health Science Center, West Yanta Street No.76, Xi’an, Shaanxi 710061, PR China