Manuel Goncalves (0000-0002-8111-1073)

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I am a molecular biologist focusing on the investigation of gene delivery and gene editing systems. After a post-graduation period in a gene therapy company (Crucell B.V.), I held a Ph.D. fellowship from the Portuguese Foundation for Science and Technology to perform research at the Faculty of Medicine of the University of Leiden (The Netherlands), on the investigation of viral vector systems for the stable genetic modification of human cells. In 2015, I became associate professor at the Department of Cell and Chemical Biology of the Leiden University Medical Center (LUMC). In this capacity, I supervise a team whose research interests are: (i) exploring viral vectors as delivery agents for gene-editing purposes; (ii) studying the role of epigenetic mechanisms on the performance of different gene-editing tools and strategies; and (iii) improving the precision of gene-editing approaches based on using programmable nucleases and ‘nickases’ alone or together with exogenous (donor) DNA constructs. In this context, our team has pioneered investigations on double strand DNA break-free gene targeting; and on establishing viral vectors as delivery vehicles of transcription activator-like effector (TALE) nucleases and clustered regularly interspaced short palindromic repeat (CRISPR) nucleases.
Main research interests: Developing and integrating gene delivery and gene editing platforms. Investigating genome editing strategies for achieving seamless and scarless genetic modification of human stem/progenitor cells for disease modelling and correction.
Lab information and activities are available via the link: https://ccb.lumc.nl/about-the-goncalves-lab-135

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Universiteit Leiden: Leiden, Zuid-Holland, NL

Associate professor (Cell and Chemical Biology - Leiden University Medical Centre)

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Manuel Goncalves

https://orcid.org/0000-0002-8111-1073

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Universiteit Leiden: Leiden, Zuid-Holland, NL

Education and qualifications (1)

Universidade de Lisboa Faculdade de Ciências: Lisbon, PT

Works (50 of 62)

Precision genome editing using combinatorial viral vector delivery of CRISPR-Cas9 nucleases and donor DNA constructs

On RNA-programmable gene modulation as a versatile set of principles targeting muscular dystrophies

Selection-free precise gene repair using high-capacity adenovector delivery of advanced prime editing systems rescues dystrophin synthesis in DMD muscle cells

“Soft” genome editing using CRISPR nickases as a potential source of safer cell products

AAV-vectored base editor trans-splicing delivers dystrophin repair

Efficient and scalable generation of primordial germ cells in 2D culture using basement membrane extract overlay

Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants

High-capacity adenovector delivery of forced CRISPR-Cas9 heterodimers fosters precise chromosomal deletions in human cells

Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells

Broadening the reach and investigating the potential of prime editors through fully viral gene-deleted adenoviral vector delivery

A primer to gene therapy: Progress, prospects, and problems

Precise and broad scope genome editing based on high-specificity Cas9 nickases.

Novel Therapeutic Approaches for the Treatment of Retinal Degenerative Diseases: Focus on CRISPR/Cas-Based Gene Editing

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

Precise and non-disruptive gene editing based on programmable nickases

Adenoviral Vectors Meet Gene Editing: A Rising Partnership for the Genomic Engineering of Human Stem Cells and Their Progeny

High-Capacity Adenoviral Vectors Permit Robust and Versatile Testing of DMD Gene Repair Tools and Strategies in Human Cells

Expanding the editable genome and CRISPR–Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking

Intronic SMCHD1 variants in FSHD: testing the potential for CRISPR-Cas9 genome editing

The Chromatin Structure of CRISPR-Cas9 Target DNA Controls the Balance between Mutagenic and Homology-Directed Gene-Editing Events

DNA, RNA, and Protein Tools for Editing the Genetic Information in Human Cells

The Chromatin Structure Differentially Impacts High-Specificity CRISPR-Cas9 Nuclease Strategies.

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting

Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes.

The emerging role of viral vectors as vehicles for DMD gene editing.

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells.

Probing the impact of chromatin conformation on genome editing tools.

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations.

Engineered Viruses as Genome Editing Devices

Genome editing at the crossroads of delivery, specificity, and fidelity

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

Adenoviral vector DNA for accurate genome editing with engineered nucleases

Construction and characterization of adenoviral vectors for the delivery of TALENs into human cells

Lentiviral vectors encoding zinc-finger nucleases specific for the model target locus HPRT1

Development of an AdEasy-based system to produce first- and second-generation adenoviral vectors with tropism for CAR- or CD46-positive cells

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

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

Histone deacetylase inhibition rescues gene knockout levels achieved with integrase-defective lentiviral vectors encoding zinc-finger nucleases

Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination

Adenoviral Vectors Stimulate Glucagon Transcription in Human Mesenchymal Stem Cells Expressing Pancreatic Transcription Factors

Concerted nicking of donor and chromosomal acceptor DNA promotes homology-directed gene targeting in human cells

Myogenic properties of human mesenchymal stem cells derived from three different sources

Nonspaced inverted DNA repeats are preferential targets for homology-directed gene repair in mammalian cells

Long-term contribution of human bone marrow mesenchymal stromal cells to skeletal muscle regeneration in mice

Transcription factor rational design improves directed differentiation of human mesenchymal stem cells into skeletal myocytes

Rapid and sensitive lentivirus vector-based conditional gene expression assay to monitor and quantify cell fusion activity

Stimulation of homology-directed gene targeting at an endogenous human locus by a nicking endonuclease

Genetic complementation of human muscle cells via directed stem cell fusion

Targeted chromosomal insertion of large DNA into the human genome by a fiber-modified high-capacity adenovirus-based vector system

Adenovirus: From foe to friend

Peer review (23 reviews for 6 publications/grants)