Programming gene and engineered-cell therapies with synthetic biology

Cas13b Is a Type VI-B CRISPR-Associated RNA-Guided RNase Differentially Regulated by Accessory Proteins Csx27 and Csx28

(2017) Aaron A. Smargon et al.   MOLECULAR CELL

RNA targeting with CRISPR–Cas13

(2017) Omar O. Abudayyeh et al.   NATURE

Programmable base editing of A•T to G•C in genomic DNA without DNA cleavage

(2017) Nicole M. Gaudelli et al.   NATURE

Epic $12 billion deal and FDA's approval raise CAR-T to new heights

(2017) Elie Dolgin   NATURE BIOTECHNOLOGY

RNA editing with CRISPR-Cas13

(2017) David B. T. Cox et al.   SCIENCE

Nucleic acid detection with CRISPR-Cas13a/C2c2

(2017) Jonathan S. Gootenberg et al.   SCIENCE

Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells

(2017) Waseem Qasim et al.   Science Translational Medicine

Gene Therapy for “Bubble Boy” Disease

(2016) Jonathan Hoggatt   CELL

Precision Tumor Recognition by T Cells With Combinatorial Antigen-Sensing Circuits

(2016) Kole T. Roybal et al.   CELL

Engineering Customized Cell Sensing and Response Behaviors Using Synthetic Notch Receptors

(2016) Leonardo Morsut et al.   CELL

Engineering T Cells with Customized Therapeutic Response Programs Using Synthetic Notch Receptors

(2016) Kole T. Roybal et al.   CELL

A synthetic biology-based device prevents liver injury in mice

(2016) Peng Bai et al.   JOURNAL OF HEPATOLOGY

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage

(2016) Alexis C. Komor et al.   NATURE

Targeted DNA demethylation in vivo using dCas9–peptide repeat and scFv–TET1 catalytic domain fusions

(2016) Sumiyo Morita et al.   NATURE BIOTECHNOLOGY

Prospects for gene-engineered T cell immunotherapy for solid cancers

(2016) Christopher A Klebanoff et al.   NATURE MEDICINE

A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition

(2016) Elizabeth A Traxler et al.   NATURE MEDICINE

Improving the efficacy and safety of biologic drugs with tolerogenic nanoparticles

(2016) Takashi K. Kishimoto et al.   Nature Nanotechnology

The roles of RNA processing in translating genotype to phenotype

(2016) Kassie S. Manning et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Genome editing using CRISPR-Cas9 to create the HPFH genotype in HSPCs: An approach for treating sickle cell disease and β-thalassemia

(2016) Lin Ye et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Synthetic gene network restoring endogenous pituitary–thyroid feedback control in experimental Graves’ disease

(2016) Pratik Saxena et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Continuous genetic recording with self-targeting CRISPR-Cas in human cells

(2016) S. D. Perli et al.   SCIENCE

 -cell-mimetic designer cells provide closed-loop glycemic control

(2016) M. Xie et al.   SCIENCE

C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector

(2016) Omar O. Abudayyeh et al.   SCIENCE

Genetic circuit design automation

(2016) A. A. K. Nielsen et al.   SCIENCE

A programmable synthetic lineage-control network that differentiates human IPSCs into glucose-sensitive insulin-secreting beta-like cells

(2016) Pratik Saxena et al.   Nature Communications

A CRISPR-based approach for targeted DNA demethylation

(2016) Xingxing Xu et al.   Cell Discovery

Targeted HIV-1 Latency Reversal Using CRISPR/Cas9-Derived Transcriptional Activator Systems

(2016) Julia K. Bialek et al.   PLoS One

Human whole-blood culture system for ex vivo characterization of designer-cell function

(2015) Lina Schukur et al.   BIOTECHNOLOGY AND BIOENGINEERING

Prosthetic gene networks as an alternative to standard pharmacotherapies for metabolic disorders

(2015) Boon Chin Heng et al.   CURRENT OPINION IN BIOTECHNOLOGY

An overview of FDA-approved biologics medicines

(2015) Michael S. Kinch   DRUG DISCOVERY TODAY

A fit-for-purpose strategy for the risk-based immunogenicity testing of biotherapeutics: a European industry perspective

(2015) Cathelijne Kloks et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Psoriasis

(2015) Wolf-Henning Boehncke et al.   LANCET

Use of miRNA Response Sequences to Block Off-target Replication and Increase the Safety of an Unattenuated, Glioblastoma-targeted Oncolytic HSV

(2015) Lucia Mazzacurati et al.   MOLECULAR THERAPY

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

Directed differentiation of cholangiocytes from human pluripotent stem cells

(2015) Mina Ogawa et al.   NATURE BIOTECHNOLOGY

Mammalian synthetic circuits with RNA binding proteins for RNA-only delivery

(2015) Liliana Wroblewska et al.   NATURE BIOTECHNOLOGY

Modular construction of mammalian gene circuits using TALE transcriptional repressors

(2015) Yinqing Li et al.   Nature Chemical Biology

Tunable and reversible drug control of protein production via a self-excising degron

(2015) Hokyung K Chung et al.   Nature Chemical Biology

Cas9 gRNA engineering for genome editing, activation and repression

(2015) Samira Kiani et al.   NATURE METHODS

Organs-on-chips at the frontiers of drug discovery

(2015) Eric W. Esch et al.   NATURE REVIEWS DRUG DISCOVERY

Remote control of therapeutic T cells through a small molecule-gated chimeric receptor

(2015) C.-Y. Wu et al.   SCIENCE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

Implantable synthetic cytokine converter cells with AND-gate logic treat experimental psoriasis

(2015) Lina Schukur et al.   Science Translational Medicine

Modularity, context-dependence, and insulation in engineered biological circuits

(2015) Domitilla Del Vecchio   TRENDS IN BIOTECHNOLOGY

Spinal muscular atrophy phenotype is ameliorated in human motor neurons by SMN increase via different novel RNA therapeutic approaches

(2015) Monica Nizzardo et al.   Scientific Reports

RNA Targeting by the Type III-A CRISPR-Cas Csm Complex of Thermus thermophilus

(2014) Raymond H.J. Staals et al.   MOLECULAR CELL

Programmable RNA Shredding by the Type III-A CRISPR-Cas System of Streptococcus thermophilus

(2014) Gintautas Tamulaitis et al.   MOLECULAR CELL

A Synthetic Multifunctional Mammalian pH Sensor and CO2 Transgene-Control Device

(2014) David Ausländer et al.   MOLECULAR CELL

Programmable RNA recognition and cleavage by CRISPR/Cas9

(2014) Mitchell R. O’Connell et al.   NATURE

Digital switching in a biosensor circuit via programmable timing of gene availability

(2014) Nicolas Lapique et al.   Nature Chemical Biology

Principles of genetic circuit design

(2014) Jennifer A N Brophy et al.   NATURE METHODS

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

Gene Editing of CCR5 in Autologous CD4 T Cells of Persons Infected with HIV

(2014) Pablo Tebas et al.   NEW ENGLAND JOURNAL OF MEDICINE

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance

(2014) Roberto A. Maldonado et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Accurate Predictions of Genetic Circuit Behavior from Part Characterization and Modular Composition

(2014) Noah Davidsohn et al.   ACS Synthetic Biology

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

(2013) Martin A Maier et al.   MOLECULAR THERAPY

The potential for manipulating RNA with pentatricopeptide repeat proteins

(2013) Yusuke Yagi et al.   PLANT JOURNAL

Reward-based hypertension control by a synthetic brain-dopamine interface

(2013) K. Rossger et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

A closed-loop synthetic gene circuit for the treatment of diet-induced obesity in mice

(2013) Katrin Rössger et al.   Nature Communications

High-throughput assessment of microRNA activity and function using microRNA sensor and decoy libraries

(2012) Gavriel Mullokandov et al.   NATURE METHODS

Foundations for the design and implementation of synthetic genetic circuits

(2012) Adrian L. Slusarczyk et al.   NATURE REVIEWS GENETICS

B-cell depletion and remissions of malignancy along with cytokine-associated toxicity in a clinical trial of anti-CD19 chimeric-antigen-receptor-transduced T cells

(2011) J. N. Kochenderfer et al.   BLOOD

Computational design approaches and tools for synthetic biology

(2011) James T. MacDonald et al.   Integrative Biology

Efficient construction of sequence-specific TAL effectors for modulating mammalian transcription

(2011) Feng Zhang et al.   NATURE BIOTECHNOLOGY

Small-molecule displacement of a cryptic degron causes conditional protein degradation

(2011) Kimberly M Bonger et al.   Nature Chemical Biology

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

(2011) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Synthetic Biology Moving into the Clinic

(2011) W. C. Ruder et al.   SCIENCE

Multi-Input RNAi-Based Logic Circuit for Identification of Specific Cancer Cells

(2011) Z. Xie et al.   SCIENCE

Feedback Control of Protein Expression in Mammalian Cells by Tunable Synthetic Translational Inhibition

(2011) James A. Stapleton et al.   ACS Synthetic Biology

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

(2010) Marina Cavazzana-Calvo et al.   NATURE

Self-sufficient control of urate homeostasis in mice by a synthetic circuit

(2010) Christian Kemmer et al.   NATURE BIOTECHNOLOGY

Stem cells to treat muscular dystrophies – Where are we?

(2010) Jinhong Meng et al.   NEUROMUSCULAR DISORDERS

Enhanced Weight Loss Following Coadministration of Pramlintide With Sibutramine or Phentermine in a Multicenter Trial

(2010) Louis J. Aronne et al.   Obesity

Genetic control of mammalian T-cell proliferation with synthetic RNA regulatory systems

(2010) Y. Y. Chen et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

PUF proteins: repression, activation and mRNA localization

(2010) Tara Quenault et al.   TRENDS IN CELL BIOLOGY

Progress and prospects: Biological properties and technological advances of herpes simplex virus type 1-based amplicon vectors

(2009) A L Epstein   GENE THERAPY

Synthetic translational regulation by an L7Ae–kink-turn RNP switch

(2009) Hirohide Saito et al.   Nature Chemical Biology

Comparative Proteomic Phenotyping of Cell Lines and Primary Cells to Assess Preservation of Cell Type-specific Functions

(2008) Cuiping Pan et al.   MOLECULAR & CELLULAR PROTEOMICS

Chemical control of protein stability and function in living mice

(2008) Laura A Banaszynski et al.   NATURE MEDICINE

Adalimumab therapy for moderate to severe psoriasis: A randomized, controlled phase III trial

(2007) Alan Menter et al.   JOURNAL OF THE AMERICAN ACADEMY OF DERMATOLOGY