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A Brazilian patient with late infantile metachromatic leukodystrophy treated with lentiviral hematopoietic stem-cell gene therapy: A report from prenatal diagnosis to early treatment

2023; Elsevier BV; Volume: 138; Issue: 2 Linguagem: Inglês

10.1016/j.ymgme.2022.107094

1096-7206

Larissa Faqueti, Gabrielle Iop, Layzon Antonio Lemos da Silva, Francyne Kubaski, Henrique B.L. Borges, Alice Brinckmann Oliveira Netto, Ana Carolina Brusius‐Facchin, Sandra Leistner‐Segal, Rejane Gus, Maria Teresa Vieira Sanseverino, Kristiane Michelin‐Tirelli, Fernanda Bender Pasetto, Fernanda Medeiros Sebastiao, Thiago Oliveira Silva, José Antonio Azevedo Magalhães, Carolina Fischinger Moura de Souza, Salvatore Recupero, Valeria Calbi, Francesca Fumagalli, Alessandro Aiuti, Roberto Giugliani,

Cytomegalovirus and herpesvirus research

DPPC and DPPC:SM large unilamellar vesicles (LUVs), prepared by extrusion, readily respond to osmotic shocks (hypo- and hyper-osmotic) by water influx/efflux (evaluated by changes in turbidity) and by entrapped calcein liberation (measured by an increase in dye fluorescence intensity). On the other hand, small unilamellar vesicles (SUVs) prepared by sonication are almost osmotically insensitive. LUVs water transport, both in hypo- and hyper-osmotic conditions, takes place faster than calcein ejection towards the external solvent. Similarly, response to a hypotonic imbalance is faster than that associated to a hypertonic stress. This difference is particularly noticeable for the increase in calcein fluorescence intensity and can be related to the large reorganization of the bilayer needed to form pores and/or to adsorb the dye to the inner leaflet of the vesicle after water efflux. Conversely, addition of SM to the vesicles barely modify the rate of calcein permeation across the bilayer.