Targeted nonviral delivery of genome editors in vivo

CA Tsuchida, KM Wasko… - Proceedings of the …, 2024 - National Acad Sciences
Cell-type-specific in vivo delivery of genome editing molecules is the next breakthrough that
will drive biological discovery and transform the field of cell and gene therapy. Here, we …

Current limitations of gene therapy for rare pediatric diseases: Lessons learned from clinical experience with AAV vectors

A Gardin, G Ronzitti - Archives de Pédiatrie, 2023 - Elsevier
Gene therapy using adeno-associated viral (AAV) vectors is a promising therapeutic strategy
for multiple inherited diseases. Following intravenous injection, AAV vectors carrying a copy …

[HTML][HTML] Challenges and Considerations of Preclinical Development for iPSC-Based Myogenic Cell Therapy

C Sun, C Serra, BH Kalicharan, J Harding, M Rao - Cells, 2024 - mdpi.com
Cell therapies derived from induced pluripotent stem cells (iPSCs) offer a promising avenue
in the field of regenerative medicine due to iPSCs' expandability, immune compatibility, and …

Muscle-targeted nanoparticles strengthen the effects of small-molecule inhibitors in ameliorating sarcopenia

J Wang, Y He, B Wang, R Yin, B Chen, H Wang - Nanomedicine, 2023 - Taylor & Francis
Background: Sarcopenia is an aging-related degeneration of muscle mass and strength.
Small-molecule inhibitor SW033291 has been shown to attenuate muscle atrophy. Targeted …

[HTML][HTML] Skeletal muscle-directed gene therapy: hijacking the fusogenic properties of muscle cells

H Büning, M Morgan, A Schambach - Signal Transduction and Targeted …, 2023 - nature.com
A recent study published in Cell by the Millay lab reports on an elegant strategy to expand
the repertoire of lentiviral (LV) vectors from being the main delivery tool for ex vivo gene …

[HTML][HTML] Muscle fusogens go viral for gene delivery to skeletal muscle

DE Gibbs, AD Pyle - Cell, 2023 - Elsevier
Viruses and multinucleated cells rely on fusogens to facilitate the fusion of their membranes.
In this issue of Cell, Millay and colleagues demonstrate that replacing viral fusogens with …

Molecular regulation of myocyte fusion.

TJ Wherley, S Thomas, DP Millay… - Current Topics in …, 2024 - europepmc.org
Myocyte fusion is a pivotal process in the development and regeneration of skeletal muscle.
Failure during fusion can lead to a range of developmental as well as pathological …

[PDF][PDF] Reg. SAL.: 2024005103

CDEF DE - 2024 - servicios.farmaceuticosdesevilla.es
El 25 de abril de 1953 se publicaba en la prestigiosa revista Nature un artículo denominado
“Estructura molecular de los ácidos nucleicos; una estructura para el ácido …