High-throughput 5′ UTR engineering for enhanced protein production in non-viral gene therapies

Comparison of Integrases Identifies Bxb1-GA Mutant as the Most Efficient Site-Specific Integrase System in Mammalian Cells

(2019) Barbara Jusiak et al.   ACS Synthetic Biology

Human 5′ UTR design and variant effect prediction from a massively parallel translation assay

(2019) Paul J. Sample et al.   NATURE BIOTECHNOLOGY

A high-throughput screening and computation platform for identifying synthetic promoters with enhanced cell-state specificity (SPECS)

(2019) Ming-Ru Wu et al.   Nature Communications

Achieving High-Yield Production of Functional AAV5 Gene Delivery Vectors via Fedbatch in an Insect Cell-One Baculovirus System

(2019) Pranav R.H. Joshi et al.   Molecular Therapy-Methods & Clinical Development

Nonviral Delivery Systems for Cancer Gene Therapy: Strategies and Challenges

(2018) Gayong Shim et al.   CURRENT GENE THERAPY

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN

(2018) Christian Hinderer et al.   HUMAN GENE THERAPY

Arginase I mRNA therapy – A novel approach to rescue arginase 1 enzyme deficiency

(2018) Kirtika H. Asrani et al.   RNA Biology

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Toward Predictable 5′UTRs in Saccharomyces cerevisiae: Development of a yUTR Calculator

(2018) Thomas Decoene et al.   ACS Synthetic Biology

Pharmaceutical Development of AAV-Based Gene Therapy Products for the Eye

(2018) Gerard A. Rodrigues et al.   PHARMACEUTICAL RESEARCH

Engineering the 5′ UTR-Mediated Regulation of Protein Abundance in Yeast Using Nucleotide Sequence Activity Relationships

(2018) Wentao Ding et al.   ACS Synthetic Biology

Plasmid DNA Manufacturing for Indirect and Direct Clinical Applications

(2017) Marco Schmeer et al.   HUMAN GENE THERAPY

A Novel DNA Vaccine Platform Enhances Neo-antigen-like T Cell Responses against WT1 to Break Tolerance and Induce Anti-tumor Immunity

(2017) Jewell N. Walters et al.   MOLECULAR THERAPY

A platform for functional assessment of large variant libraries in mammalian cells

(2017) Kenneth A. Matreyek et al.   NUCLEIC ACIDS RESEARCH

Synthetic recombinase-based state machines in living cells

(2016) N. Roquet et al.   SCIENCE

Synthetic biology and microbioreactor platforms for programmable production of biologics at the point-of-care

(2016) Pablo Perez-Pinera et al.   Nature Communications

Clinical development of gene therapy: results and lessons from recent successes

(2016) Sandeep RP Kumar et al.   Molecular Therapy-Methods & Clinical Development

Massively parallel high-order combinatorial genetics in human cells

(2015) Alan S L Wong et al.   NATURE BIOTECHNOLOGY

DNA vaccines, electroporation and their applications in cancer treatment

(2015) Si-Hyeong Lee et al.   Human Vaccines & Immunotherapeutics

Translation of 5′ leaders is pervasive in genes resistant to eIF2 repression

(2015) Dmitry E Andreev et al.   eLife

Identifying and engineering promoters for high level and sustainable therapeutic recombinant protein production in cultured mammalian cells

(2014) Steven C. L. Ho et al.   BIOTECHNOLOGY LETTERS

Translation from a DMD exon 5 IRES results in a functional dystrophin isoform that attenuates dystrophinopathy in humans and mice

(2014) Nicolas Wein et al.   NATURE MEDICINE

Ribosome profiling: new views of translation, from single codons to genome scale

(2014) Nicholas T. Ingolia   NATURE REVIEWS GENETICS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Clearance of persistent HPV infection and cervical lesion by therapeutic DNA vaccine in CIN3 patients

(2014) Tae Jin Kim et al.   Nature Communications

CCL21 Cancer Immunotherapy

(2014) Yuan Lin et al.   Cancers

Vascular Endothelial Growth Factor and Angiogenesis in the Regulation of Cutaneous Wound Repair

(2014) Kelly E. Johnson et al.   Advances in Wound Care

Chromatin Position Effects Assayed by Thousands of Reporters Integrated in Parallel

(2013) Waseem Akhtar et al.   CELL

Translational repression using BIV Tat peptide–TAR RNA interaction in mammalian cells

(2013) Chaitanya Sudrik et al.   CHEMICAL COMMUNICATIONS

Rapid, modular and reliable construction of complex mammalian gene circuits

(2013) Patrick Guye et al.   NUCLEIC ACIDS RESEARCH

Deciphering the rules by which 5'-UTR sequences affect protein expression in yeast

(2013) S. Dvir et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Genome-Scale CRISPR-Cas9 Knockout Screening in Human Cells

(2013) Ophir Shalem et al.   SCIENCE

Optimized and enhanced DNA plasmid vector based in vivo construction of a neutralizing anti-HIV-1 envelope glycoprotein Fab

(2013) Kar Muthumani et al.   Human Vaccines & Immunotherapeutics

The translational landscape of mTOR signalling steers cancer initiation and metastasis

(2012) Andrew C. Hsieh et al.   NATURE

Synergistic antitumoral effect of combination E gene therapy and Doxorubicin in MCF-7 breast cancer cells

(2011) Ana R. Rama et al.   BIOMEDICINE & PHARMACOTHERAPY

Gene therapy finds its niche

(2011) Cormac Sheridan   NATURE BIOTECHNOLOGY

Lentiviral Vectors in Gene Therapy: Their Current Status and Future Potential

(2010) David Escors et al.   ARCHIVUM IMMUNOLOGIAE ET THERAPIAE EXPERIMENTALIS

Construction of a novel expression cassette for increasing transgene expression in vivo in endothelial cells of large blood vessels

(2010) N Dronadula et al.   GENE THERAPY

Serine recombinases as tools for genome engineering

(2010) William R.A. Brown et al.   METHODS

Evaluating post-transcriptional regulatory elements for enhancing transient gene expression levels in CHO K1 and HEK293 cells

(2009) Mariati et al.   PROTEIN EXPRESSION AND PURIFICATION

Genome-Wide Analysis in Vivo of Translation with Nucleotide Resolution Using Ribosome Profiling

(2009) N. T. Ingolia et al.   SCIENCE