Srivalli TelikepalliHeather E. ShinoglePrem S. ThapaJae Hyun KimMeghana DeshpandeVibha JawaC. Russell MiddaughLinda NarhiMarisa K. JoubertDavid B. Volkin
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An IgG2 monoclonal antibody (mAb) solution was subjected to stirring, generating high concentrations of nanometer and subvisible particles, which were then successfully size-enriched into different size bins by low-speed centrifugation or a combination of gravitational sedimentation and fluorescence-activated cell sorting (FACS). The size-fractiona...
Gene therapy is a promising approach for the treatment of a variety of disorders including genetic diseases and cancer. Among the viral vectors used in gene therapy, the lentiviral vector, based on HIV-1, is the only integrative vector able to transduce nondividing cells. The first generation of lentiviral vector was established in 1996. Since then...
p53 and p63 share extensive sequence and structure homology. p53 is frequently mutated in cancer, whereas mutations in p63 cause developmental disorders manifested in ectodermal dysplasia, limb defects, and orofacial clefting. We have established primary adult skin keratinocytes from ectrodactyly, ectodermal dysplasia, and cleft lip/palate (EEC) sy...
Among the key properties that distinguish adult mammalian stem cells from their more differentiated progeny is the ability of stem cells to remain in a quiescent state for prolonged periods of time 1,2 . However, the molecular pathways for the maintenance of stem cell quiescence remain elusive. Using adult muscle stem cells (" satellite cells " (SC...
A mixed-chimerism approach is a major goal to circumvent sustained immunosuppression, but most of the proposed protocols need antibody treatment or host irradiation. Another promising experience involves bu-sulfan combined with cyclophosphamide treatment. Additionally, recent publications demonstrated that, differing from busulfan, treosulfan admin...
Duchenne muscular dystrophy (DMD) is an X-linked genetic disease characterized by the absence of dystrophin (427 kDa). An approach to eventually restore this protein in patients with DMD is to introduce into their muscles a plasmid encoding dystrophin cDNA. Because the phenotype of the dystrophic dog is closer to the human phenotype than is the mdx...
