Publicaties
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Hyper-functional coagulation factor IX improves the efficacy of gene therapy in hemophilic mice. Vrije Universiteit Brussel
Gene therapy may provide a cure for hemophilia and overcome the limitations of protein replacement therapy. Increasing the potency of gene transfer vectors may allow to improve their therapeutic index, as lower doses can be administered to achieve therapeutic benefit, reducing toxicity of in vivo administration. Here we generated codon-usage optimized and hyper-functional factor IX (FIX) transgenes carrying a R338L amino acid substitution, ...
Developing nanobodies to advance immune checkpoint therapy that targets lymphocyteactivation gene 3 Vrije Universiteit Brussel
The activation of immune cells is controlled by a plethora of mechanisms. For
example, inhibitory immune checkpoints (ICPs) are imperative to control and limit
T-cell activation and functionality. These control mechanisms can prevent excessive inflammation, tissue damage and ultimately autoimmunity. Unfortunately, cancer cells exploit ICPs to generate additional defense mechanisms against the host’s immune system. Upregulation of ...
example, inhibitory immune checkpoints (ICPs) are imperative to control and limit
T-cell activation and functionality. These control mechanisms can prevent excessive inflammation, tissue damage and ultimately autoimmunity. Unfortunately, cancer cells exploit ICPs to generate additional defense mechanisms against the host’s immune system. Upregulation of ...
Gene therapy for allergic diseases Universiteit Gent
On the cellular processing of nanomedicines for retinal gene therapy and their suitability for intravitreal injections Universiteit Gent
The 1st WFH Gene Therapy Round Table Vrije Universiteit Brussel
In this first in a series of round table meetings, the 1st World Federation of Hemophilia Gene Therapy Round Table was convened to initiate a global dialogue on the expected challenges and opportunities that a disruptive therapy, such as gene therapy, will bring to the haemophilia community. Perspectives from key stakeholder groups, including healthcare professionals, regulators, payors, people with hemophilia and pharmaceutical industry ...
Gene Therapy For Hemophilia B Using CB 2679d-GT: A Novel Factor IX Variant With Higher Potency Than Factor IX Padua Vrije Universiteit Brussel KU Leuven
Sustained expression of therapeutic factor IX (FIX) levels has been achieved after adeno-associated viral (AAV) vector-based gene therapy in patients with hemophilia B. Nevertheless, patients are still at risk of vector dose-limiting toxicity, particularly liver inflammation justifying the need for more efficient vectors and a lower dosing regimen. A novel increased potency FIX (designated as CB 2679d-GT), containing three amino acid ...
Genome-Wide Computational Analysis Reveals Cardiomyocyte-Specific Transcriptional CIS-Regulatory Motifs that Enable Efficient Cardiac Gene Therapy. Vrije Universiteit Brussel KU Leuven Universiteit Gent
Gene therapy is a promising emerging therapeutic modality for the treatment of cardiovascular diseases and hereditary diseases that afflict the heart. Hence, there is a need to develop robust cardiac-specific expression modules that allow for stable expression of the gene of interest in cardiomyocytes. We therefore explored a new approach based on a genome-wide bio-informatics strategy that revealed novel cardiac-specific cis-acting regulatory ...
Liver-Specific Transcriptional Modules Identified by Genome-Wide in Silico Analysis Enable Efficient Gene Therapy in Mice and Non-Human Primates. Vrije Universiteit Brussel
The robustness and safety of liver-directed gene therapy can be substantially improved by enhancing expression of the therapeutic transgene in the liver. To achieve this, we developed a new approach of rational in silico vector design. This approach relies on a genome-wide bio-informatics strategy to identify cis-acting regulatory modules (CRMs) containing evolutionary conserved clusters of transcription factor binding site motifs that determine ...
Recent developments in transposon-mediated gene therapy. Vrije Universiteit Brussel
INTRODUCTION: The continuous improvement of gene transfer technologies has broad implications for stem cell biology, gene discovery, and gene therapy. Although viral vectors are efficient gene delivery vehicles, their safety, immunogenicity and manufacturing challenges hamper clinical progress. In contrast, non-viral gene delivery systems are less immunogenic and easier to manufacture. AREAS COVERED: In this review, we explore the emerging ...