Kyriel M. Pineault
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View article: Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy
Pharmacological and pre-clinical safety profile of rSIV.F/HN, a hybrid lentiviral vector for cystic fibrosis gene therapy Open
Rationale and objective Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. CFTR modulators offer significant improvements, but ∼10% of patients remain nonresponsive or are intolerant. Thi…
View article: Challenges in Development and Qualification of PCR/dPCR Assays for Gene Therapy Biodistribution and Viral Shedding Assessment
Challenges in Development and Qualification of PCR/dPCR Assays for Gene Therapy Biodistribution and Viral Shedding Assessment Open
Gene therapies are part of a larger class of advanced therapies that aim to treat disease via delivery of recombinant genetic material. A gene therapy product has two components, the delivery system (viral vector or non-viral) and the tran…
View article: Squamous cell carcinoma subverts adjacent histologically normal epithelium to promote lateral invasion
Squamous cell carcinoma subverts adjacent histologically normal epithelium to promote lateral invasion Open
Recurrent and new tumors, attributed in part to lateral invasion, are frequent in squamous cell carcinomas and lead to poor survival. We identified a mechanism by which cancer subverts adjacent histologically normal epithelium to enable sm…
View article: <i>Hox</i> genes maintain critical roles in the adult skeleton
<i>Hox</i> genes maintain critical roles in the adult skeleton Open
Significance Mammalian Hox genes are absolutely critical for proper development of skeletal morphology during embryogenesis, and Hox11 genes are essential for proper zeugopod (radius/ulna, tibia/fibula) development. While the expression of…
View article: Meeting Report: Instituto Gulbenkian de Ci&ecirc;ncia-Institut Curie Young Scientists' Retreat 2019
Meeting Report: Instituto Gulbenkian de Ciência-Institut Curie Young Scientists' Retreat 2019 Open
This paper report presents the interdisciplinary science showcased at the Young Scientists Retreat 2019. The retreat was jointly organized by young researchers from Instituto Gulbenkian de Ciência (Portugal) and Institut Curie (Franc…
View article: Hox11 expressing regional skeletal stem cells are progenitors for osteoblasts, chondrocytes and adipocytes throughout life
Hox11 expressing regional skeletal stem cells are progenitors for osteoblasts, chondrocytes and adipocytes throughout life Open
Multipotent mesenchymal stromal cells (MSCs) are required for skeletal formation, maintenance, and repair throughout life; however, current models posit that postnatally arising long-lived adult MSCs replace transient embryonic progenitor …
View article: Bone morphology is regulated modularly by global and regional genetic programs
Bone morphology is regulated modularly by global and regional genetic programs Open
Bone protrusions provide stable anchoring sites for ligaments and tendons and define the unique morphology of each long bone. Despite their importance, the mechanism by which superstructures are patterned is unknown. Here, we identify comp…
View article: Bone Morphology is Regulated Modularly by Global and Regional Genetic Programs
Bone Morphology is Regulated Modularly by Global and Regional Genetic Programs Open
During skeletogenesis, a variety of protrusions of different shapes and sizes develop on the surfaces of long bones. These superstructures provide stable anchoring sites for ligaments and tendons during the assembly of the musculoskeletal …
View article: <i>Hox11</i> Function Is Required for Region-Specific Fracture Repair
<i>Hox11</i> Function Is Required for Region-Specific Fracture Repair Open
The processes that govern fracture repair rely on many mechanisms that recapitulate embryonic skeletal development. Hox genes are transcription factors that perform critical patterning functions in regional domains along the axial and limb…