A graduate of the Faculty of Medicine of Uruguay, Dr. Daniel Skuk worked for six years as a neuromuscular pathologist in his home country. He moved to Quebec in 1996 to work with Dr. Jacques P Tremblay’s team, focusing on research in cell therapy and regenerative medicine in skeletal muscle. Since 1997, he is the only researcher to use nonhuman primates (NHP) in this field. Through his NHP studies, Dr. Skuk designed the transplant technique that allowed Dr. Tremblay’s team to develop new clinical trials in patients with Duchenne muscular dystrophy (DMD). In these trials, Dr. Skuk applied his transplant protocol to patients, obtaining the best confirmed results for cell therapy in the muscle. He was invited to the Nice Reference Center for Neuromuscular Diseases to teach his technique for a clinical trial on fascio-scapulohumeral muscular dystrophy. Dr. Skuk was also the only Canadian researcher to take part in expert meetings organized by the French Association against Myopathies to evaluate the future of cell therapy in this field.
Regenerative medicine and cell therapy for degenerative and/or genetic disorders of skeletal muscle.
Muscular dystrophies are characterized by muscle degeneration of genetic origin. Due to its relative frequency and very severe prognosis – muscle degeneration in the limbs and trunk during childhood and adolescence leading to an almost complete loss of motor skills and premature death due to cardiorespiratory complications – most of the research efforts area focused on DMD, to treat these pathologies. The molecular basis of DMD is the absence of the dystrophin protein, which causes repeated necrosis of the myofibers until their regenerative capacity is exhausted, leading to an almost complete loss of muscle tissue, which is replaced by fibrosis and adipose tissue. The treatments for muscular dystrophies must therefore have three objectives: to correct the molecular defect in as many myofibers as necessary, to form new myofibers, and to provide new stem cells to maintain the regeneration capacity. Correction of the molecular defect as a therapeutic target is shared by gene therapy and cell therapy, while other objectives are exclusive to cell therapy and regenerative medicine. By prioritizing NHP – whose phylogenetic proximity to humans makes them crucial for preclinical research – Dr. Skuk’s research aims at developing effective protocols for this clinical application of cell therapy, as well as understanding and defining several aspects of clinical relevance in this context.
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Myotubes Formed De Novo by Myoblasts Injected into the Scar of Myocardial Infarction Persisted for 16 Years in a Patient: Importance for Regenerative Medicine in Degenerative Myopathies.Journal Article
Stem Cells Transl Med, 8 (3), pp. 313-314, 2019, ISSN: 2157-6564.
Sarcolemmal Complement Membrane Attack Complex Deposits During Acute Rejection of Myofibers in Nonhuman Primates.Journal Article
J Neuropathol Exp Neurol, 78 (1), pp. 38-46, 2019, ISSN: 0022-3069.
Nonfunctional mutant Wrn protein leads to neurological deficits, neuronal stress, microglial alteration, and immune imbalance in a mouse model of Werner syndrome.Journal Article
Brain Behav Immun, 73 , pp. 450-469, 2018, ISSN: 0889-1591.
De Novo Circulating Antidonor's Cell Antibodies During Induced Acute Rejection of Allogeneic Myofibers in Myogenic Cell Transplantation: A Study in Nonhuman Primates.Journal Article
Transplant Direct, 3 (12), pp. e228, 2017, ISSN: 2373-8731.
Cell Therapy in Myology: Dynamics of Muscle Precursor Cell Death after Intramuscular Administration in Non-human Primates.Journal Article
Mol Ther Methods Clin Dev, 5 , pp. 232-240, 2017, ISSN: 2329-0501.
CD56+ Muscle Derived Cells but Not Retinal NG2+ Perivascular Cells of Nonhuman Primates are Myogenic after Intramuscular Transplantation in Immunodeficient MiceJournal Article
J Stem Cell Res Ther, 7 (2), 2017.
The Process of Engraftment of Myogenic Cells in Skeletal Muscles of Primates: Understanding Clinical Observations and Setting Directions in Cell Transplantation Research.Journal Article
Cell Transplant, 26 (11), pp. 1763-1779, 2017, ISSN: 0963-6897.
Confirmation of donor-derived dystrophin in a duchenne muscular dystrophy patient allotransplanted with normal myoblasts.Journal Article
Muscle Nerve, 54 (5), pp. 979-981, 2016, ISSN: 0148-639X.
Cell therapy in muscular dystrophies: many promises in mice and dogs, few facts in patients.Journal Article
Expert Opin Biol Ther, 15 (9), pp. 1307-19, 2015, ISSN: 1471-2598.
Thérapie cellulaire et génique (Chapitre 7)Book Chapter
P, Beaulieu; V, Pichette; J, Desroches; Souich, Du (Ed.): Précis de pharmacologie : du fondamental à la clinique, pp. 161-182, Montréal, QC, Les Presses de l'Université de Montréal, 2015, ISBN: 9782760634527.