A5012946265- CRISPR and Genetic Engineering
- Pluripotent Stem Cells Research
- Virus-based gene therapy research
- Animal Virus Infections Studies
- SARS-CoV-2 and COVID-19 Research
- Erythrocyte Function and Pathophysiology
- Wnt/β-catenin signaling in development and cancer
- Hemoglobinopathies and Related Disorders
- RNA Interference and Gene Delivery
- HIV Research and Treatment
- COVID-19 Clinical Research Studies
- Neuroinflammation and Neurodegeneration Mechanisms
- Renal Diseases and Glomerulopathies
- Lysosomal Storage Disorders Research
- Hippo pathway signaling and YAP/TAZ
- Autism Spectrum Disorder Research
- Viral gastroenteritis research and epidemiology
- Protein Degradation and Inhibitors
- Retinoids in leukemia and cellular processes
- Autophagy in Disease and Therapy
- PARP inhibition in cancer therapy
- Immunotherapy and Immune Responses
- Innovation and Socioeconomic Development
- Genetic and Kidney Cyst Diseases
- Prenatal Screening and Diagnostics
Mahidol University
2012-2024
Inserm
2019-2022
Institut des Maladies Génétiques Imagine
2019-2022
Université Paris Cité
2016-2022
Sorbonne Paris Cité
2022
Centre National de la Recherche Scientifique
2019-2021
Laboratory of Excellence GR-Ex
2019-2021
Assistance Publique – Hôpitaux de Paris
2021
Sorbonne Université
2021
Groupe de Recherches et d'Echanges Technologiques
2021
Abstract Gaucher disease (GD) is a lysosomal storage disorder caused by mutation in the GBA1 gene, responsible for encoding enzyme Glucocerebrosidase (GCase). Although neuronal death and neuroinflammation have been observed brains of individuals with neuronopathic (nGD), exact mechanism underlying neurodegeneration nGD remains unclear. In this study, we used two induced pluripotent stem cells (iPSCs)-derived cell lines acquired from type-3 GD patients (GD3-1 GD3-2) to investigate mechanisms...
Abstract Hematopoietic stem-cell (HSC) transplantation using a donor with homozygous mutation in the HIV co-receptor CCR5 (CCR5Δ32/Δ32) holds great promise as cure for HIV-1. Previously, there were three patients that had been reported to be completely cured from infection by this approach. However, finding naturally suitable Human Leukocyte Antigen (HLA)-matched CCR5Δ32 is very difficult. The prevalence of allele only 1% Caucasian population. Therefore, additional sources CCR5Δ32/Δ32 HSCs...
Abstract We have reported that of the 10 commonly used AAV serotype vectors, AAV6 is most efficient in transducing primary human hematopoietic stem/progenitor cells (HSPCs). However, transduction efficiency wild-type (WT) vector varies greatly HSPCs from different donors. Here we report two distinct strategies to further increase donors are transduced less efficiently with WT vectors. The first strategy involved modifications viral capsid proteins where specific surface-exposed tyrosine (Y)...
Updated and revised versions of COVID-19 vaccines are vital due to genetic variations the SARS-CoV-2 spike antigen. Furthermore, that safe, cost-effective, logistic-friendly critically needed for global equity, especially middle- low-income countries. Recombinant protein-based subunit against have been reported using receptor-binding domain (RBD) prefusion trimers (S-2P). Recently, a new version trimers, named HexaPro, has shown possess two RBD in “up” conformation, its physical property, as...
Although gene transfer to hematopoietic stem cells (HSCs) has shown therapeutic efficacy in recent trials for several individuals with inherited disorders, transduction incompleteness of the HSC population remains a hurdle yield cure all patients reasonably low integrated vector numbers. In previous attempts at selection, massive loss transduced HSCs, contamination non-transduced cells, or lack applicability large cell populations rendered procedures out reach human applications. Here, we...
Gaucher disease (GD) is one of the most prevalent lysosomal storage diseases caused by mutation glucocerebrosidase (GBA1) gene. GD patients develop symptoms in various organs body; however, underlying mechanisms causing pathology are still elusive. Thus, a suitable model important order to facilitate subsequent investigations. Here, we established MUi031-A human induced pluripotent stem cell (hiPSC) line from CD34+ hematopoietic cells female type-3 patient with homozygous c.1448 T > C...
The diarylheptanoid ASPP 049 has improved the quality of adult hematopoietic stem cell (HSC) expansion ex vivo through long-term reconstitution in animal models. However, its effect on regeneration from human induced pluripotent cells (hiPSCs) is unknown. We utilized a defined cocktail cytokines without serum or feeder followed by supplementation to produce stem/progenitor (HSPCs). Flow cytometry and trypan blue exclusion analysis were used identify nonadherent adherent cells. Nonadherent...
Human immunodeficiency virus (HIV)-1 infection is an important public health problem worldwide. After primary HIV-1 infection, transcribed DNA integrated into the host genome, serving as a reservoir of and hindering definite cure. Although highly active antiretroviral therapy suppresses viral replication, resulting in undetectable levels HIV RNA blood, rebound can be detected after few weeks treatment interruption. This supports concept that there stable people living with HIV-1. Recently,...
Gambogic acid (GA) has been reported to induce apoptosis in cholangiocarcinoma (CCA) cell lines. However, the molecular mechanisms underlying its anti-cancer activity remain poorly understood. This study was aimed investigate GA's effect on human CCA lines, KKU-M213 and HuCCA-1, associated Wnt/β-catenin signaling pathway.Cell viability, apoptosis, cycle analysis were conducted by MTT flow cytometry. The of GA mediated ER stress determined luciferase-reporter assay, qRT-PCR, western blot...
Targeting immune checkpoints, such as Programmed cell Death 1 (PD1), has improved survival in cancer patients by restoring antitumor responses. Most patients, however, relapse or are refractory to checkpoint blocking therapies. Neuropilin-1 (NRP1) is a transmembrane glycoprotein required for nervous system and angiogenesis embryonic development, also expressed cells. We hypothesized that NRP1 could be an co-receptor modulating CD8+ T cells activity the context of response. Here, we show...
Gaucher disease (GD) is a common lysosomal storage resulting from mutations in the glucocerebrosidase (GBA1) gene. This genetic disorder manifests with symptoms affecting multiple organs, yet underlying mechanisms leading to pathology remain elusive. In this study, we successfully generated MUi030-A human induced pluripotent stem cell (hiPSC) line using non-integration method male type-3 GD patient homozygous c.1448T>C (L444P) mutation. These hiPSCs displayed normal karyotype and...
Abstract Updated and revised versions of COVID-19 vaccines are vital due to genetic variations the SARS-CoV-2 spike antigen. Furthermore, that safe, cost-effective, logistically friendly critically needed for global equity, especially middle low income countries. Recombinant protein-based subunit against have been reported with use receptor binding domain (RBD) prefusion trimers (S-2P). Recently, a new version trimers, so called “HexaPro”, has shown its physical property possess two RBD in...
Hematopoietic stem cells (HSCs, CD34+ cells) have shown therapeutic efficacy for transplantation in various hematological disorders. However, a large quantity of HSCs is required transplantation. Therefore, strategies to increase HSC numbers and preserve functions through ex vivo culture are critically required. Here, we report that expansion medium supplemented with ASPP 049, diarylheptanoid isolated from Curcuma comosa, cocktail cytokines markedly increased adult cells. Interestingly,...
Hematopoietic stem and progenitor cell (HSPC) transplantation is a curative treatment of hematological disorders that has been utilized for several decades. Although umbilical cord blood (UCB) promising source HSPCs, the low dose HSPCs in these preparations limits their use, prompting need ex vivo HSPC expansion. To establish more efficient method to expand UCB we developed bioactive peptide named SL-13R cultured (CD34+ cells) with animal component-free medium containing cytokine cocktail....