Research group - Samir EL Andaloussi
Our research efforts focus on one of the greatest challenges facing modern therapeutics; delivery. Despite the unprecedented knowledge of diseases and their mechanisms due to advances in biomedical sciences; many promising therapeutic approaches are still clinically unavailable. This is simply because there is no efficient means of delivering these therapeutics to the right organ with the right dose.
Such therapeutics include proteins for replacement therapy or antibodies for anti-inflammatory treatment as well as nucleic acids for gene therapy of diseases including muscular dystrophies and neurodegenerative disorders. We develop innovative drug delivery technologies that are able to carry protein and/or gene therapies to the target tissues safely and efficiently. We have developed a promising technology based on reprogramming exosomes, which are vesicles that are naturally used by cells to communicate, to carry therapeutic proteins and nucleic acids. We engineer exosomes with enhanced delivery and targeting capacities and screen our wide array of exosome designs in disease-relevant cellular and animal models. We are also developing methods for large-scale production and purification of such exosome therapeutics. Additionally, our lab develops methods for gene therapy based on modified cell-penetrating peptides that are efficient carriers of nucleic acids and proteins across biological barriers. We test our different technologies in models of inflammatory diseases as well as neurodegenerative and neuromuscular disorders. Our main aim is to one day unlock the potential of protein and gene therapy of such diseases by the development of novel and efficient delivery technologies.
Research group leader - Samir EL Andaloussi
|Osama Ahmed||Graduate Student|
|Safa Safin Hayder Bazaz Hidush||Graduate Student|
|Giulia Corso||Graduate Student, Technical assistant|
|Dhanu Gupta||PhD student|
|Oskar Gustafsson||R&D trainee|
|André Görgens||Associated, Postdoc|
|Doste Rashid Mamand||Graduate Student|
|Oscar Wiklander||Graduate Student|
Swedish Medical Research Council, EuroNanoMed (EraNet), Swedish Society of Medical Research (SSMF), KID funding, Vinnova, Moderna Therapeutics, Åke Wiberg stiftelse, SSF IRC funding, Evox Therapeutics, KI Senior Researcher central funding
Functional Delivery of Lipid-Conjugated siRNA by Extracellular Vesicles.
Mol. Ther. 2017 07;25(7):1580-1587
Exosomes surf on filopodia to enter cells at endocytic hot spots, traffic within endosomes, and are targeted to the ER.
J. Cell Biol. 2016 04;213(2):173-84
Ultrafiltration with size-exclusion liquid chromatography for high yield isolation of extracellular vesicles preserving intact biophysical and functional properties.
Nanomedicine 2015 May;11(4):879-83
Functional correction in mouse models of muscular dystrophy using exon-skipping tricyclo-DNA oligomers.
Nat. Med. 2015 Mar;21(3):270-5
Extracellular vesicle in vivo biodistribution is determined by cell source, route of administration and targeting.
J Extracell Vesicles 2015 ;4():26316
Serum-free culture alters the quantity and protein composition of neuroblastoma-derived extracellular vesicles.
J Extracell Vesicles 2015 ;4():26883
Correlating In Vitro Splice Switching Activity With Systemic In Vivo Delivery Using Novel ZEN-modified Oligonucleotides.
Mol Ther Nucleic Acids 2014 Nov;3():e212
Extracellular vesicles: biology and emerging therapeutic opportunities.
Nat Rev Drug Discov 2013 May;12(5):347-57
Exosome-mediated delivery of siRNA in vitro and in vivo.
Nat Protoc 2012 Dec;7(12):2112-26
Design of a peptide-based vector, PepFect6, for efficient delivery of siRNA in cell culture and systemically in vivo.
Nucleic Acids Res. 2011 May;39(9):3972-87
A peptide-based vector for efficient gene transfer in vitro and in vivo.
Mol. Ther. 2011 Aug;19(8):1457-67