Lipopeptide-mediated Cas9 RNP delivery: A promising broad therapeutic strategy for safely removing deep-intronic variants in ABCA4

Authors: Irene Vázquez-Domínguez, Mert Öktem, Florian A. Winkelaar, Thai Hoang Nguyen, Anita D.M. Hoogendoorn, Eleonora Roschi, Galuh D.N. Astuti, Raoul Timmermans, Nuria Suárez-Herrera, Ilaria Bruno, Albert Ruiz-Llombart, Joseph Brealey, Olivier G. de Jong, Rob W.J. Collin, Enrico Mastrobattista, Alejandro Garanto

Source: Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102345- (2024)

Subject Terms: MT: RNA/DNA Editing, peptide-mediated delivery, CRISPR-Cas9 genome editing, lipopeptide, intron removal, ABCA4 deep-intronic variants, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253124002324; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/9cd8756ec318431f8076236a1e749258

Harnessing the Power of NK Cell Receptor Engineering as a New Prospect in Cancer Immunotherapy

Authors: Stefania Douka, Vasilis Papamoschou, Monica Raimo, Enrico Mastrobattista, Massimiliano Caiazzo

Source: Pharmaceutics, Vol 16, Iss 9, p 1143 (2024)

Subject Terms: cancer immunotherapy, adoptive cell transfer, NK cells, tumor microenvironment, receptor engineering, NK cell clinical trials, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/16/9/1143; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/25440d46c8e74bb5b564a5df7c48954e

Amphipathic Cell-Penetrating Peptide-Aided Delivery of Cas9 RNP for In Vitro Gene Editing and Correction

Authors: Mert Öktem, Enrico Mastrobattista, Olivier G. de Jong

Source: Pharmaceutics, Vol 15, Iss 10, p 2500 (2023)

Subject Terms: CRISPR-Cas9, cell-penetrating peptide (CPP), LAH5, RNP, HDR, delivery, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/15/10/2500; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/bedc8eb3fd47404aac364c859c1cf902

Cas9 RNP transfection by vapor nanobubble photoporation for ex vivo cell engineering

Authors: Laurens Raes, Melissa Pille, Aranit Harizaj, Glenn Goetgeluk, Jelter Van Hoeck, Stephan Stremersch, Juan C. Fraire, Toon Brans, Olivier Gerrit de Jong, Roel Maas-Bakker, Enrico Mastrobattista, Pieter Vader, Stefaan C. De Smedt, Bart Vandekerckhove, Koen Raemdonck, Kevin Braeckmans

Source: Molecular Therapy: Nucleic Acids, Vol 25, Iss , Pp 696-707 (2021)

Subject Terms: CRISPR-Cas9, gene editing, T cells, stem cells, photoporation, intracellular delivery, Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2162253121002080; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/d83a80530b204562bf8701e1aa7bfddd

Microbubble-Assisted Ultrasound for Drug Delivery to the Retina in an Ex Vivo Eye Model

Authors: Charis Rousou, Nicky van Kronenburg, Andreas F. P. Sonnen, Marijke van Dijk, Chrit Moonen, Gert Storm, Enrico Mastrobattista, Roel Deckers

Source: Pharmaceutics, Vol 15, Iss 4, p 1220 (2023)

Subject Terms: ultrasound, microbubbles, intracellular accumulation, ex vivo perfused eye, retinal drug delivery, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/15/4/1220; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/4c3e25033dd34dc18301425251e35cd8

Inducible Fibril Formation of Silk–Elastin Diblocks

Authors: Lione Willems, Stefan Roberts, Isaac Weitzhandler, Ashutosh Chilkoti, Enrico Mastrobattista, John van der Oost, Renko de Vries

Source: ACS Omega, Vol 4, Iss 5, Pp 9135-9143 (2019)

Subject Terms: Chemistry, QD1-999

File Description: electronic resource

Relation: https://doaj.org/toc/2470-1343

Access URL: https://doaj.org/article/e8864204f8544e25b2d634dd31b719b5

The Effect of Microbubble-Assisted Ultrasound on Molecular Permeability across Cell Barriers

Authors: Charis Rousou, Josanne de Maar, Boning Qiu, Kim van der Wurff-Jacobs, Marika Ruponen, Arto Urtti, Sabrina Oliveira, Chrit Moonen, Gert Storm, Enrico Mastrobattista, Roel Deckers

Source: Pharmaceutics, Vol 14, Iss 3, p 494 (2022)

Subject Terms: ultrasound, microbubbles, epithelial permeation, paracellular permeability, intracellular accumulation, nanobody, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/14/3/494; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/617e21bbdeb14c44b399418198e81166

Impact of Formulation Conditions on Lipid Nanoparticle Characteristics and Functional Delivery of CRISPR RNP for Gene Knock-Out and Correction

Authors: Johanna Walther, Danny Wilbie, Vincent S. J. Tissingh, Mert Öktem, Heleen van der Veen, Bo Lou, Enrico Mastrobattista

Source: Pharmaceutics, Vol 14, Iss 1, p 213 (2022)

Subject Terms: CRISPR, LNP, formulation, NHEJ, HDR, AF4, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/14/1/213; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/ea9ccfd5fe4c4ad8884b10d3f129f281

Retinoic Acid-Containing Liposomes for the Induction of Antigen-Specific Regulatory T Cells as a Treatment for Autoimmune Diseases

Authors: Daniëlle ter Braake, Naomi Benne, Chun Yin Jerry Lau, Enrico Mastrobattista, Femke Broere

Source: Pharmaceutics, Vol 13, Iss 11, p 1949 (2021)

Subject Terms: tolerance, liposomes, nanoparticles, tolerogenic dendritic cells, retinoic acid, autoimmunity, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/13/11/1949; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/920c80c20edc43d18cb32ef89961f26d

Ultrasound and Microbubbles for the Treatment of Ocular Diseases: From Preclinical Research towards Clinical Application

Authors: Charis Rousou, Carl C. L. Schuurmans, Arto Urtti, Enrico Mastrobattista, Gert Storm, Chrit Moonen, Kai Kaarniranta, Roel Deckers

Source: Pharmaceutics, Vol 13, Iss 11, p 1782 (2021)

Subject Terms: ultrasound, microbubble, drug delivery, blood–retina barrier, ocular drug delivery, USMB, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/13/11/1782; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/34378b0abe5948f2ad711c4b85d6fe61

Lipid-mediated Wnt protein stabilization enables serum-free culture of human organ stem cells

Authors: Nesrin Tüysüz, Louis van Bloois, Stieneke van den Brink, Harry Begthel, Monique M. A. Verstegen, Luis J. Cruz, Lijian Hui, Luc J. W. van der Laan, Jeroen de Jonge, Robert Vries, Eric Braakman, Enrico Mastrobattista, Jan J. Cornelissen, Hans Clevers, Derk ten Berge

Source: Nature Communications, Vol 8, Iss 1, Pp 1-11 (2017)

Subject Terms: Science

File Description: electronic resource

Relation: https://doaj.org/toc/2041-1723

Access URL: https://doaj.org/article/c1491917e9fe4c3fa3f5944fd3318bf1

Light-Triggered Cellular Delivery of Oligonucleotides

Authors: Leena-Stiina Kontturi, Joep van den Dikkenberg, Arto Urtti, Wim E. Hennink, Enrico Mastrobattista

Source: Pharmaceutics, Vol 11, Iss 2, p 90 (2019)

Subject Terms: oligonucleotide delivery, light-activated release, intracellular release, liposome, indocyanine green, Pharmacy and materia medica, RS1-441

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4923/11/2/90; https://doaj.org/toc/1999-4923

Access URL: https://doaj.org/article/bc473a206b624fa5b1119834b96f4614

Wnt3a protein reduces growth factor-driven expansion of human hematopoietic stem and progenitor cells in serum-free cultures.

Authors: Lucia E Duinhouwer, Nesrin Tüysüz, Elwin W J C Rombouts, Mariette N D Ter Borg, Enrico Mastrobattista, Jan Spanholtz, Jan J Cornelissen, Derk Ten Berge, Eric Braakman

Source: PLoS ONE, Vol 10, Iss 3, p e0119086 (2015)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC4373922?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/92581bf04e164092a8c2b639f73ff7e7

Looped Structure of Flowerlike Micelles Revealed by 1H NMR Relaxometry and Light Scattering.

Authors: Albert J. de Graaf, Kristel W. M. Boere, Johan Kemmink, Remco G. Fokkink, Cornelus F. van Nostrum, Dirk T. S. Rijkers, Jasper van der Gucht, Hans Wienk, Marc Baldus, Enrico Mastrobattista, Tina Vermonden, Wim E. Hennink

Source: Langmuir; Aug2011, Vol. 27 Issue 16, p9843-9848, 6p

Conformation and Intermolecular Interactions of SA2 Peptides Self-Assembled into Vesicles.

Authors: Albert J. van Hell¹, Andrey Klymchenko¹, Pepijn P. Burgers¹, Ed E. Moret¹, Wim Jiskoot¹, Wim E. Hennink¹, Daan J. A. Crommelin¹, Enrico Mastrobattista¹

Source: Journal of Physical Chemistry B; Sep2010, Vol. 114 Issue 34, p11046-11052, 7p

Stabilization of Peptide Vesicles by Introducing Inter-Peptide Disulfide Bonds.

Authors: Albert van Hell, Daan Crommelin, Wim Hennink, Enrico Mastrobattista

Source: Pharmaceutical Research; Sep2009, Vol. 26 Issue 9, p2186-2193, 8p

Subject Terms: OLIGOPEPTIDES, CHEMICAL bonds, SULFIDES, MOLECULAR self-assembly, MOLECULE-molecule collisions, CROSSLINKING (Polymerization), AMINO acid sequence, ELECTRON microscopy

Physicochemical and Immunological Characterization of N,N,N-Trimethyl Chitosan-Coated Whole Inactivated Influenza Virus Vaccine for Intranasal Administration.

Authors: Niels Hagenaars¹, Enrico Mastrobattista¹, Rolf Verheul¹, Imke Mooren², Harrie Glansbeek³, Jacco Heldens³, Han van den Bosch³, Wim Jiskoot⁴

Source: Pharmaceutical Research. Jun2009, Vol. 26 Issue 6, p1353-1364. 12p.

Subject Terms: *CHITOSAN, *INFLUENZA vaccines, *DRUG coatings, *INTRANASAL medication, *PHARMACEUTICAL chemistry, *IMMUNOGENETICS, *HEMAGGLUTININ, *CHEMICAL inhibitors, *THERAPEUTICS

Plasmid CpG Depletion Improves Degree and Duration of Tumor Gene Expression After Intravenous Administration of Polyplexes.

Authors: Holger de Wolf, Nina Johansson, Anh-Thy Thong, Cor Snel, Enrico Mastrobattista, Wim Hennink, Gert Storm

Source: Pharmaceutical Research; Jul2008, Vol. 25 Issue 7, p1654-1662, 9p

Subject Terms: GENES, MOBILE genetic elements, TUMORS, CYSTS (Pathology)

Self-Assembly of Recombinant Amphiphilic Oligopeptides into Vesicles.

Authors: Albert J. van Hell, Cristina I. C. A. Costa, Frits M. Flesch, Marc Sutter, Wim Jiskoot, Daan J. A. Crommelin, Wim E. Hennink, Enrico Mastrobattista

Source: Biomacromolecules; Sep2007, Vol. 8 Issue 9, p2753-2761, 9p

Plasmid Engineering for Controlled and Sustained Gene Expression for Nonviral Gene Therapy.

Authors: Ethlinn van Gaal, Wim Hennink, Daan Crommelin, Enrico Mastrobattista

Source: Pharmaceutical Research; Jun2006, Vol. 23 Issue 6, p1053-1074, 22p