Jacques P. Tremblay received a B.Sc. in Biochemistry from McGill University in 1970, and a Ph.D. in Neuroscience from UCSD (University of California in San Diego) in 1974. From 1975 to 1976, he was a postdoctoral fellow at the Laboratory of Neurobiology of l’Hôpital de l’Enfant-Jésus. Subsequently, he spent his entire career at Laval University: Professor under grant from 1976 to 1981 in the Department of Anatomy; Assistant Professor from 1981 to 1985; full Professor from 1985; Director of the Department of Anatomy from 1987 to 1997, and Professor of the Department of Molecular Medicine from 2010 to now. He is currently a regular researcher at the Axis of Neuroscience of the CHU Research Center of Quebec-Université Laval.

Development of a treatment for Duchenne Muscular Dystrophy (DMD)

DMD is due to a mutation of the gene coding for the dystrophin protein. This mutation leads to an absence of this protein under the membrane of muscle fibers. His laboratory is renowned for his work on the transplantation of normal allogeneic myoblasts as a treatment for DMD. His Phase I clinical trial for this therapy showed that this transplant restores the expression of this protein in the patient’s muscle fibers. In 2006, Dr. Tremblay received the Henry Friesen Award from the Royal College of Physicians and Surgeons of Canada for his work on this therapy. His group is currently conducting a Phase I / II clinical trial on this therapy. In addition, his group is currently using CRISPR / Cas9 technology to correct the dystrophin gene, creating an additional deletion to produce a hybrid exon of the dystrophy gene, which not only restores the expression of dystrophin but also produces dystrophin with a normal structure.

Development of a treatment for Friedreich’s Ataxia

Dr. Tremblay’s group has also been conducting research on Friedreich’s Ataxia since 2010. This disease is due to an elongation of the GAA trinucleotide repeat in intron 1 of the frataxin gene, which reduces expression of this protein, leading to the death of neurons and cardiomyocytes that induce neurological and cardiac symptoms. His group demonstrated that the expression of frataxin is increased by targeting the promoter of this gene with TALE-VP64 proteins. In addition, it has also demonstrated that it is possible to suppress trinucleotide repetition by cutting with the CRISPR / Cas9 system before and after this repeat.

Development of a treatment for Alzheimer’s disease

This group also uses CRISPR / Cas9 technology to develop a treatment for Alzheimer’s disease. This disease is due to the abnormal metabolism of the APP protein (Amyloid Precursor Protein) which leads to the formation of beta-amyloid peptides that form plaques. The formation of these peptides can be greatly reduced by the A673T mutation of the APP gene observed in a small portion of Iceland’s population. Dr. Tremblay’s group has demonstrated that this mutation could be produced with the CRISPR / Cas9 system.

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Yaméogo P, Duchêne BL, Majeau N, Tremblay JP

CRISPR-SCReT (CRISPR-Stop Codon Read Through) method to control Cas9 expression for gene editing.

Journal Article

Gene Ther, 2021.

Abstract | Links:

Benabdallah B, Desaulniers-Langevin C, Goyer ML, Colas C, Maltais C, Li Y, Guimond JV, Tremblay JP, Haddad E, Beausejour C

Myogenic progenitor cells derived from human induced pluripotent stem cell are immune-tolerated in humanized mice.

Journal Article

Stem Cells Transl Med, 10 (2), 2021.

Abstract | Links:

Guyon A, Rousseau J, Begin FG, Bertin T, Lamothe G, Tremblay JP

Base editing strategy for insertion of the A673T mutation in the APP gene to prevent the development of AD .

Journal Article

Mol Ther Nucleic Acids, 24 , 2021.

Abstract | Links:

Tremblay JP, Annoni A, Suzuki M

Three Decades of Clinical Gene Therapy: From Experimental Technologies to Viable Treatments.

Journal Article

Mol Ther, 29 (2), 2021.

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Mendell JR, Al-Zaidy SA, Rodino-Klapac LR, Goodspeed K, Gray SJ, Kay CN, Boye SL, Boye SE, George LA, Salabarria S, Corti M, Byrne BJ, Tremblay JP

Current Clinical Applications of In Vivo Gene Therapy with AAVs.

Journal Article

Mol Ther, 29 (2), 2021.

Abstract | Links:

Guyon A, Rousseau J, Lamothe G, Tremblay JP

The protective mutation A673T in amyloid precursor protein gene decreases Aβ peptides production for 14 forms of Familial Alzheimer's Disease in SH-SY5Y cells.

Journal Article

PLoS One, 15 (12), 2020.

Abstract | Links:

Skuk D, Tremblay JP

A Comment on "Muscle Xenografts Reproduce Key Molecular Features of Facioscapulohumeral Muscular Dystrophy": What Is New and What Has Already been Done and Reported but Was Not Quoted?

Journal Article

Cell Transplant, 29 , 2020.

Abstract | Links:

Skuk D, Tremblay JP

Human Muscle Precursor Cells Form Human-Derived Myofibers in Skeletal Muscles of Nonhuman Primates: A Potential New Preclinical Setting to Test Myogenic Cells of Human Origin for Cell Therapy of Myopathies.

Journal Article

J Neuropathol Exp Neurol, 79 (12), 2020.

Abstract | Links:

Skuk D, Tremblay JP

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), 2019.

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Pinto M, Zorn KC, Tremblay JP, Desroches J, Dallaire F, Aubertin K, Marple E, Kent C, Leblond F, Trudel D, Lesage F

Integration of a Raman spectroscopy system to a robotic-assisted surgical system for real-time tissue characterization during radical prostatectomy procedures.

Journal Article

J Biomed Opt, 24 (2), 2019.

Abstract | Links:

278 entries « 1 of 28 »
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Active projects

  • Correction by CRISPR base editing of point mutations responsible for Duchenne Muscular Dystrophy, from 2020-06-01 to 2022-05-31
  • Correction of the c. 121 A to T mutation in the NKX6-2 gene by PRIME editing, from 2021-08-23 to 2022-08-22
  • Deciphering the role of DCIR in HIV-1 pathogenesis, from 2018-04-01 to 2023-03-31
  • Development of a rapid and simple test to detect the COVID-19 variants that can be used in remote areas and developing countries, from 2021-06-01 to 2022-05-31
  • Développement d’une thérapie génique pour l’ataxie de Friedreich, from 2021-05-31 to 2023-05-31
  • Génération de lignées isogéniques pour les mutations GNA11/BAP1 par « PRIME editing » comme modèles d’études du mélanome oculaire., from 2021-04-01 to 2022-03-31
  • Phase I/II clinical trial of myoblast transplantation to Duchenne Muscular Dystrophy patients., from 2013-10-01 to 2022-03-31
  • PRIME editing correction of the T1709M mutation responsible for some cases of Ryanodine receptor type I-related myopathies, from 2021-07-01 to 2023-06-30
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Friedreich patient cells and in the YG8sR mouse model, from 2019-10-01 to 2024-09-30
  • Utilisation de vésicules extracellulaires pour livrer les composants de la technologie d’édition du génome CRISPR/PRIME, from 2021-04-01 to 2022-03-31

Recently finished projects

  • Acquisition d'un congélateur à basse température (-80C) , from 2020-10-09 to 2021-03-31
  • Acquisition d'une centrifugeuse , from 2019-10-28 to 2020-03-31
  • Association d'hydrogel et de pré-conditionnement pharmacologique pour améliorer la greffe de myoblastes, from 2020-04-01 to 2021-03-31
  • Correction of the dystrophin gene with Zinc Finger Proteins and TAL effector nuclease, from 2012-10-01 to 2020-03-31
  • Development of a molecular therapies for Freidreich Ataxia., from 2013-01-01 to 2021-03-31
  • Development of a treatment of Alzheimer based on the editing of the Amyloid Precuror Protein gene with the CRISPR system., from 2016-01-15 to 2020-09-30
  • Les cellules souches musculaires dérivées d'un modèle de culture d'iPSC en 3D comme stratégie thérapeutique pour le traitement de la dystrophie musculaire, from 2019-04-01 to 2020-03-31
  • Phase I/II clinical trial delivering myoblasts to DMD patients, from 2019-10-31 to 2020-05-01
  • Préparation d'un document de présentation sur la recherche en génétique humaine , from 2019-11-16 to 2020-03-31
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Freidreich patient cells and in the YG8sR mouse model, from 2018-07-01 to 2020-06-30
  • Removal of the GAA repeat with the CRISPR/Cas9 system in Freidreich patient cells and in the YG8sR mouse model, from 2018-07-01 to 2020-06-30
  • Using extracellular vesicles to deliver therapeutic proteins for various ataxia, from 2020-03-01 to 2021-07-01
  • Utilisation de la technologie CRISPR / Cas9 dans le développement d'un traitement permanent contre la maladie d'Alzheimer, from 2018-04-01 to 2020-03-30
Data provided by the Université Laval research projects registery