Viral Vectors, Engineered Cells and the CRISPR Revolution (Q45874356)

17 December 2017

title

Viral Vectors, Engineered Cells and the CRISPR Revolution (English)

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17 December 2017

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James E DiCarlo

1

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17 December 2017

Anurag Deeconda

series ordinal

2

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17 December 2017

Stephen H Tsang

series ordinal

3

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17 December 2017

language of work or name

English

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publication date

1 January 2017

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17 December 2017

Advances in Experimental Medicine and Biology

published in

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17 December 2017

volume

1016

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17 December 2017

page(s)

3-27

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17 December 2017

The Significance of Pneumococcal Types

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STUDIES ON THE CHEMICAL NATURE OF THE SUBSTANCE INDUCING TRANSFORMATION OF PNEUMOCOCCAL TYPES: INDUCTION OF TRANSFORMATION BY A DESOXYRIBONUCLEIC ACID FRACTION ISOLATED FROM PNEUMOCOCCUS TYPE III

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Genetics of human cess line. IV. DNA-mediated heritable transformation of a biochemical trait

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Applications of CRISPR technologies in research and beyond

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Malignant Transformation of Cells by Viruses

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Double-strand break end resection and repair pathway choice

21 January 2018

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Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes

21 January 2018

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The delitto perfetto approach to in vivo site-directed mutagenesis and chromosome rearrangements with synthetic oligonucleotides in yeast

21 January 2018

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Homologous recombination in plant cells is enhanced by in vivo induction of double strand breaks into DNA by a site-specific endonuclease

21 January 2018

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Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease

21 January 2018

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In vivo genome editing as a potential treatment strategy for inherited retinal dystrophies

21 January 2018

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Chimeric restriction enzymes: what is next?

21 January 2018

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Hybrid restriction enzymes: zinc finger fusions to Fok I cleavage domain

21 January 2018

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ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

21 January 2018

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Highly efficient endogenous human gene correction using designed zinc-finger nucleases

21 January 2018

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A novel zinc-finger nuclease platform with a sequence-specific cleavage module.

21 January 2018

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Breaking the code of DNA binding specificity of TAL-type III effectors

21 January 2018

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TAL effectors are remote controls for gene activation

21 January 2018

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Targeting DNA double-strand breaks with TAL effector nucleases

21 January 2018

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A TALE nuclease architecture for efficient genome editing

21 January 2018

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Differential integrity of TALE nuclease genes following adenoviral and lentiviral vector gene transfer into human cells.

21 January 2018

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Nucleotide sequence of the iap gene, responsible for alkaline phosphatase isozyme conversion in Escherichia coli, and identification of the gene product

21 January 2018

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Unusual nucleotide arrangement with repeated sequences in the Escherichia coli K-12 chromosome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Biological significance of a family of regularly spaced repeats in the genomes of Archaea, Bacteria and mitochondria.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Evolution and classification of the CRISPR-Cas systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Identification of a novel family of sequence repeats among prokaryotes

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The CRISPR system: small RNA-guided defense in bacteria and archaea

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Structures of Cas9 Endonucleases Reveal RNA-Mediated Conformational Activation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

An updated evolutionary classification of CRISPR-Cas systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

RNA-guided human genome engineering via Cas9

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Multiplex genome engineering using CRISPR/Cas systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Fanconi anemia gene editing by the CRISPR/Cas9 system

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene therapy in patient-specific stem cell lines and a preclinical model of retinitis pigmentosa with membrane frizzled-related protein defects

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Precision Medicine: Genetic Repair of Retinitis Pigmentosa in Patient-Derived Stem Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A Combinatorial CRISPR-Cas9 Attack on HIV-1 DNA Extinguishes All Infectious Provirus in Infected T Cell Cultures

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

CRISPR/Cas9-Mediated Genome Editing of Herpesviruses Limits Productive and Latent Infections

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A CRISPR Way to Block PERVs--Engineering Organs for Transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Genome-wide inactivation of porcine endogenous retroviruses (PERVs).

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

New CRISPR-Cas systems from uncultivated microbes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Multiplex gene editing by CRISPR-Cpf1 using a single crRNA array

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Engineered CRISPR-Cas9 nucleases with altered PAM specificities

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

CRISPR knockout screening outperforms shRNA and CRISPRi in identifying essential genes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Rationally engineered Cas9 nucleases with improved specificity

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Structures of a CRISPR-Cas9 R-loop complex primed for DNA cleavage

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

GUIDE-seq enables genome-wide profiling of off-target cleavage by CRISPR-Cas nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Cas9 gRNA engineering for genome editing, activation and repression.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Highly efficient Cas9-mediated transcriptional programming

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Repurposing the CRISPR-Cas9 system for targeted DNA methylation

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Integrase-defective lentiviral vectors as a delivery platform for targeted modification of adenosine deaminase locus

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Cellular immune responses to HIV.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Altering the tropism of lentiviral vectors through pseudotyping

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Redirecting retroviral tropism by insertion of short, nondisruptive peptide ligands into envelope

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Antibody-mediated targeting of replication-competent retroviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Lentivirus pre-packed with Cas9 protein for safer gene editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Novel lentiviral vectors with mutated reverse transcriptase for mRNA delivery of TALE nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Unraveling CRISPR-Cas9 genome engineering parameters via a library-on-library approach

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Harnessing the CRISPR/Cas9 system to disrupt latent HIV-1 provirus

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The therapeutic application of CRISPR/Cas9 technologies for HIV.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

CRISPR/Cas9 cleavage of viral DNA efficiently suppresses hepatitis B virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Histone deacetylase inhibition activates transgene expression from integration-defective lentiviral vectors in dividing and non-dividing cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A modified γ-retrovirus vector for X-linked severe combined immunodeficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Progress and challenges in viral vector manufacturing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Chimeric antigen receptor T cells for sustained remissions in leukemia

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenoviruses: update on structure and function

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenovirus: the first effective in vivo gene delivery vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenoviruses as gene/vaccine delivery vectors: promises and pitfalls

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Engineered Viruses as Genome Editing Devices

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Development of Novel Adenoviral Vectors to Overcome Challenges Observed With HAdV-5-based Constructs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenovirus vectors for gene therapy, vaccination and cancer gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenovirus-mediated in vivo gene transfer and expression in normal rat liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Efficient and accurate homologous recombination in hESCs and hiPSCs using helper-dependent adenoviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Targeted gene correction of laminopathy-associated LMNA mutations in patient-specific iPSCs

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Progress and prospects: zinc-finger nucleases as gene therapy agents

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenoviral vector delivery of RNA-guided CRISPR/Cas9 nuclease complexes induces targeted mutagenesis in a diverse array of human cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenoviral vector DNA for accurate genome editing with engineered nucleases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Establishment of HIV-1 resistance in CD4 T cells by genome editing using zinc-finger nucleases

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Targeted genome correction by a single adenoviral vector simultaneously carrying an inducible zinc finger nuclease and a donor template.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Capsid-modified adenoviral vectors for improved muscle-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Effect of adenovirus serotype 5 fiber and penton modifications on in vivo tropism in rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Immunity to adenovirus and adeno-associated viral vectors: implications for gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene therapy: trials and tribulations

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A human type 5 adenovirus-based tuberculosis vaccine induces robust T cell responses in humans despite preexisting anti-adenovirus immunity

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Safety and Immunogenicity of a rAd35-EnvA Prototype HIV-1 Vaccine in Combination with rAd5-EnvA in Healthy Adults (VRC 012)

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adenovirus-Vectored Broadly Neutralizing Antibodies Directed Against gp120 Prevent Human Immunodeficiency Virus Type 1 Acquisition in Humanized Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Disrupted adenovirus-based vaccines against small addictive molecules circumvent anti-adenovirus immunity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A phase I trial of intravenous CG7870, a replication-selective, prostate-specific antigen-targeted oncolytic adenovirus, for the treatment of hormone-refractory, metastatic prostate cancer.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Oncolytic adenovirus-mediated therapy for prostate cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Therapy of prostate cancer using a novel cancer terminator virus and a small molecule BH-3 mimetic

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

ADENOVIRUS-ASSOCIATED DEFECTIVE VIRUS PARTICLES

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Systemic delivery of adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Integration of adeno-associated virus (AAV) and recombinant AAV vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

In vivo tissue-tropism of adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Organization of the adeno-associated virus (AAV) capsid gene: mapping of a minor spliced mRNA coding for virus capsid protein 1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A viral assembly factor promotes AAV2 capsid formation in the nucleolus

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene therapy for the CNS using AAVs: The impact of systemic delivery by AAV9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adeno-associated virus type 2 contains an integrin alpha5beta1 binding domain essential for viral cell entry

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The 37/67-kilodalton laminin receptor is a receptor for adeno-associated virus serotypes 8, 2, 3, and 9

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

An essential receptor for adeno-associated virus infection.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A multifunctional AAV-CRISPR-Cas9 and its host response

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

AAV-Mediated CRISPR/Cas Gene Editing of Retinal Cells In Vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Immune responses to AAV in clinical trials

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Innate Immune Responses to AAV Vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Efficient and Targeted Transduction of Nonhuman Primate Liver With Systemically Delivered Optimized AAV3B Vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Unique glycan signatures regulate adeno-associated virus tropism in the developing brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Controlling AAV Tropism in the Nervous System with Natural and Engineered Capsids.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A phase I study of an adeno-associated virus-CFTR gene vector in adult CF patients with mild lung disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Long-term safety and efficacy of factor IX gene therapy in hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Long-term effect of gene therapy on Leber's congenital amaurosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Efficacy and long-term safety of alipogene tiparvovec (AAV1-LPLS447X) gene therapy for lipoprotein lipase deficiency: an open-label trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

$1-million price tag set for Glybera gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adoptive transfer of costimulated CD4 T cells induces expansion of peripheral T cells and decreased CCR5 expression in HIV infection.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Restoration of immunity in lymphopenic individuals with cancer by vaccination and adoptive T-cell transfer

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Development of immuno-oncology drugs - from CTLA4 to PD1 to the next generations

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Adoptive cellular therapy: a race to the finish line.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The cytoplasmic domain of the T cell receptor zeta chain is sufficient to couple to receptor-associated signal transduction pathways

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Chimeric receptors with 4-1BB signaling capacity provoke potent cytotoxicity against acute lymphoblastic leukemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Chimeric receptors containing CD137 signal transduction domains mediate enhanced survival of T cells and increased antileukemic efficacy in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

In vivo persistence, tumor localization, and antitumor activity of CAR-engineered T cells is enhanced by costimulatory signaling through CD137 (4-1BB)

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Genetically targeted T cells eradicate systemic acute lymphoblastic leukemia xenografts

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

CD28 costimulation provided through a CD19-specific chimeric antigen receptor enhances in vivo persistence and antitumor efficacy of adoptively transferred T cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Eradication of systemic B-cell tumors by genetically targeted human T lymphocytes co-stimulated by CD80 and interleukin-15.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

T-cell clones can be rendered specific for CD19: toward the selective augmentation of the graft-versus-B-lineage leukemia effect

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Blood cell-derived induced pluripotent stem cells free of reprogramming factors generated by Sendai viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The Oct4 and Nanog transcription network regulates pluripotency in mouse embryonic stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Induction of pluripotent stem cells from adult human fibroblasts by defined factors

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

The Combination of CRISPR/Cas9 and iPSC Technologies in the Gene Therapy of Human β-thalassemia in Mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-Derived Muscle Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Functional Correction of Large Factor VIII Gene Chromosomal Inversions in Hemophilia A Patient-Derived iPSCs Using CRISPR-Cas9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Playing the end game: DNA double-strand break repair pathway choice

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Small molecules enhance CRISPR genome editing in pluripotent stem cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Gene Conversion Tracts from Double-Strand Break Repair in Mammalian Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

Site-specific cleavage of DNA–RNA hybrids by zinc finger/FokI cleavage domain fusions

21 January 2018

1 reference

https://api.crossref.org/works/10.1007/978-3-319-63904-8_1

10.1007/978-3-319-63904-8_1

21 January 2018

Identifiers

DOI

1 reference

17 December 2017

1 reference

17 December 2017