
Department of Medical Oncology (Cancer Center Amsterdam)
Alternative splicing in lung cancer, and generation and validation of oncolytic adenoviruses based on screening data.
Group members: T. Brachtlová, J. Li, I. Milenova, I. va der Meulen-Muileman
Key words: Oncolytic adenoviruses; Therapeutic target discovery; RNA interference; Drug development
https://research.vumc.nl/en/persons/victor-w-van-beusechem; http://www.orca-therapeutics.nl/
Department of Experimental Cardiology (Heart Failure Research Center)
Development of adeno-associated virus-based gene therapy for restoring heart rhythm .
Group members:
Key words: Basic research; Translational research; Viral vectors; Cell engineering; Gene replacement.

Department of Viroscience
Developing the Newcastle Disease virus for use in viro-immuno-therapy for patients with pancreatic cancer.
Group members:
Key words: Basic research; Translational research; Oncolytic viruses; Viral vectors; Immunity.
https://www.erasmusmc.nl/viroscience/research/researchers/vandenhoogen/
Department of Neurosurgery
Exploring extracellular vesicles (exosomes) as a tool to improve (gene) therapy or diagnosis of cancer
Group members: M. Lamfers, E. Stavrakaki, L. Vogelezang
Key words: Gene Therapy; Oncolytic viruses; Exosomes; Extracellular vesicle; Cancer.

Department of Hematology
Development and implementation of T cell receptor gene therapy for hematological malignancies and solid tumors
Key words: Translational research; Viral vectors; Immunity; Gene replacement.
Department of Cell and Chemical Biology
Development of virus-derived tools for gene and stem-cell therapy. We evaluate our vectors in collaboration with clinically oriented groups in a variety of disease models.
Group members: Dr. V. Kemp
Key words: Basic research; Translational research; Oncolytic viruses; Viral vectors; Immunity; Regenerative medicine; Cell engineering; Genome editing/repair; Gene replacement.

Department of Prescision Medicine M-Lab
Development of novel and innovative therapies for cancer treatment and vaccination based on the use of prokaryotic living medicine vectors.
Group members: Prof. Dr. P. Lambin, Dr. L. Dubois, Dr. T. Bailey, Dr. A. Kubiak, Dr. A. Mowday, N. Lieuwes, R. Biemans.
Key words: Translational research; Living medicine; prokaryotic vectors; cancer; vaccination.

Department of Human Genetics
Development of therapeutic molecular strategies to correct genetic defects causing inherited retinal diseases.
Group members: Dr. I. Vázquez Domínguez, L. Duijkers, T. Tomkiewicz, M. Peeters, N. Suárez Herrera
Key words: Basic research; Translational research; Viral vectors; Cell engineering; Genome editing/repair; Gene replacement; RNA therapeutics; inherited retinal diseases.
Department of Pediatrics (Amalia Children’s Hospital) & Department of Human Genetics
Development of novel therapeutic strategies at DNA and RNA level to correct genetic defects causing neurometabolic or inherited retinal diseases.
Group members: Dr. I. Vázquez Domínguez, A. Hoogendoorn, L. Duijkers, T.Tomkiewicz, N. Suárez Herrera, I. Schuurmans
Key words: Basic research; Translational research; Viral vectors; Cell engineering; Genome editing/repair; Gene replacement; RNA therapeutics; inherited retinal diseases; inherited neurometabolic diseases.
Department of Cell Biology
Development of improved delivery strategies (in vitro and in vivo) for therapeutic antisense oligonucleotides in the context of Myotonic dystrophy type 1.
Group members:
Key words: Translational research; RNA therapeutics.

Department of Medical Microbiology
Development of strategies for the immunotherapeutic treatment of virus-induced cancer.
Group members:
Key words: Basic research; Translational research; Viral vectors; Vaccines; Immunity.
Department of Chemical and Pharmaceutical Biology
Enhancing the efficacy and specificity of targeted genome editing with CRISPR/CAS9.
Group members: C. Cheng, D. Cheng, P. Ettema, F.J. Dekker
Key words: CRISPR/CAS9, genome editing

Department of Orthopaedics
Biological regeneration of joint tissues; the development of (one-stage) cell or cell-based therapies to repair cartilage and meniscus tissue and restore joint homeostasis.
Group members:
Key words: Translational research; Exosomes; Regenerative medicine; Cell therapy.

Department of Pharmacology
One of the things we work on is the evaluation of new therapies for genetic kidney diseases like cystinosis. We use human proximal tubular cells with different gene mutations to study the molecular pathways affected and see how we can use gene editing to restore the gene defect.
Group members:
Key words: Translational research; Regenerative medicine; Cell engineering; Genome editing/repair; Gene replacement.
Department of Pharmaceutics
Design and pre-clinical testing of tailor-made drug delivery systems for site-specific, time- and/or rate-controlled delivery of small drugs, therapeutic proteins, nucleic acids, and antigens for use in diseases such as cancer, inflammation and infectious diseases.
Group members:
Key words: Basic research; Translational research; Vaccines; Genome editing/repair; Synthetic vectors; RNA therapeutics.
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