Exon skipping for 6 and 7 exons of DMD gene (preclinical)
TargetHeart: delivery of morpholinos to heart (discovery)
Antisense morpholino oligos
Marlin Biotech has built its own facility to produce GMP grade morpholino antisense molecules.
GENOME EDITING with CRISPR/Cas9
Marlin Biotech has successfully generated a custom model of DMD mouse with exons 8-34 deleted with CRISPR/Cas9 system.
AAV based gene delivery
We focus on AAV based gene delivery as AAVs look as most promising vehicle to target human cells.
Reshetov denis Scientific director
Dr. Denis Reshetov specializes in the field of evolutionary genomics and molecular genetics. His work is aimed at studying the genetic basis of rare genetic diseases and aging mechanisms, as well as the evolution of humans and animals.
Denis Reshetov participated in the development of new technologies for genome sequencing and analysis of epigenetic profile of a man.
Vlodavets Dmitry Medical director
Dr. Dmitry Vlodavets is director of Russian Neuromuscular Center. His work is connected with diagnosis of neuro-musclular diseases and clinical trials in this field.
(our team members are highlighted)
1. Carlier Pierre G,..., Vlodavets, Dmitry, Skeletal Muscle Quantitative Nuclear Magnetic Resonance Imaging and Spectroscopy as an Outcome Measure for Clinical Trials, Journal of Neuromuscular Diseases, vol. 3, no. 1, pp. 1-28, 2016
2. Vlodavets D., Reshetov D., S. Artemieva, E. Litvinova, I. Shulyakova, O. Shidlovskaya, D. Kazakov, E. Belousova, Russian experience of DMD genetic database, Neuromuscular Disorders 2016 October 8.
3. Dimitrieva TV, Reshetov DA, Zhernovkov VE, Vlodavets DV, Zotova ED, Ermolkevich TG, Deykin AV, Modification of the method for analysis of genome editing results using CRISPR/Cas9 system on preimplantation mouse embryos. JRSMU 2016 June 21.
4. Vlodavets D., Reshetov D., Open access cryobank of primary cell cultures from Duchenne muscular dystrophy patients, Proceeding of XII CONGRESS OF MEDITERRANEAN SOCIETY OF MYOLOGY Naples, 2015 May 15.
5. Moroz LL, … Reshetov D et al. The ctenophore genome and the evolutionary origins of neural systems (2014) Nature; 510(7503): 109-14.
6. Zotova ED, Reshetov DA, Zhernovkov VE, Vlodavets DV, Dimitrieva TV, Deykin AV, Analysis of phenotype expressions of deletions in the dystrophin gene in terms of efficiency of exon skipping as a method for treatment of hereditary dystrophinopathies. JRSMU 2016 June 21.
7. Andreeva TV, ... Reshetov DA et al. Whole exome sequencing links dental tumor to an autosomal-dominant mutation in ANO5 gene associated with gnathodiaphyseal dysplasia and muscle dystrophies. Sci Rep. 2016 May 24.
8. Protasova MS, ... Reshetov DA et al. Whole-genome sequencing identifies a novel ABCB7 gene mutation for X-linked congenital cerebellar ataxia in a large family of Mongolian ancestry. Eur J Hum Genet. 2015 Aug 5.
9. Shulha HP, Crisci JL, Reshetov D et al. Human-specific histone methylation signatures at transcription start sites in prefrontal neurons. PLoS Biol. 2012; 10(11): e1001427.
10. Vassiliev I, Vassilieva S, Harrison SJ, Nottle MB. Isolation and in vitro characterization of putative porcine embryonic stem cells from cloned embryos treated with trichostatin A. Cellular Reprogramming. 06/2011; 13(3):205‐13
11. Gursky Y., ... Deikin A. et al. Expression of fulllength human prourokinase in mammary glands of transgenic mice. Transgenic Research. 05/2009; 18(5):747‐56
12. Vassilieva S, Goh H, Lau K, Hughes J, Familari M, Rathjen P, Rathjen J. A system to enrich for primitive streak‐derivatives, definitive endoderm and mesoderm, from pluripotent cells in culture. PLoS ONE 01/2012; 7(6):e38645
2016/07/05 - Marlin Biotech got a resident status of Skolkovo Innovation Center.
2016/01/18 - Marlin Biotech launched a service for animal and cell models creation with CRISPR/Cas9.
2015/12/05 - Marlin Biotech cryobank of primary cell cultures from patients with Duchenne muscular dystrophy got 100th cell line!
2015/11/04 - First 5 mice with deletion of exons 8-34 were born in Marlin Biotech trasgenic mice facility.
2015/07/08 - Marlin Biotech validated internal CRISPR/Cas9 protocol for genome edited mice model creation.
2015/02/16 - Marlin Biotech submitted documents to achieve Skolkovo Innovation Center resident status.
2014/09/15 - M29 was found to be non-toxic at 12 mg/kg dose.
2014/09/09 - Marlin Biotech created a cryobank of primary cell cultures from patients with Duchenne muscular dystrophy. More than 30 cell lines are collected to the date.
2014/07/13 - First toxicology experiments with M29 have started.
2014/05/23 - We have finished screening of antisense oligonucleotides for exon 6 and 7 skipping at DMD gene. M29 drug candidate was selected for following development.