T
Terence R. Flotte
Researcher at University of Massachusetts Medical School
Publications - 338
Citations - 20618
Terence R. Flotte is an academic researcher from University of Massachusetts Medical School. The author has contributed to research in topics: Adeno-associated virus & Genetic enhancement. The author has an hindex of 74, co-authored 325 publications receiving 19037 citations. Previous affiliations of Terence R. Flotte include Case Western Reserve University & National Institutes of Health.
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Journal ArticleDOI
Treatment of Leber Congenital Amaurosis Due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results of a Phase I Trial
William W. Hauswirth,Tomas S. Aleman,Shalesh Kaushal,Artur V. Cideciyan,Sharon B. Schwartz,Lili Wang,Thomas J. Conlon,Sanford L. Boye,Terence R. Flotte,Barry J. Byrne,Samuel G. Jacobson +10 more
TL;DR: A recombinant adeno-associated virus serotype 2 vector, altered to carry the human RPE65 gene (rAAV2-CBSB-hRPE65) restored vision in animal models with R PE65 deficiency, and Comparisons are drawn between the present work and two other studies of ocular gene therapy for RPE 65-LCA that were carried out contemporaneously and reported.
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Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
Artur V. Cideciyan,Tomas S. Aleman,Sanford L. Boye,Sharon B. Schwartz,Shalesh Kaushal,Alejandro J. Roman,Jijing Pang,Alexander Sumaroka,Elizabeth A. M. Windsor,James M. Wilson,Terence R. Flotte,Gerald A. Fishman,Elise Heon,Edwin M. Stone,Barry J. Byrne,Samuel G. Jacobson,William W. Hauswirth +16 more
TL;DR: It is found that the intervention could overcome nearly all of the loss of light sensitivity resulting from the biochemical blockade, however, this reconstituted retinoid cycle was not completely normal.
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Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector.
Terence R. Flotte,Sandra Afione,Carol Conrad,Sharon A. McGrath,Rikki Solow,Hidemi Oka,Pamela L. Zeitlin,William B. Guggino,Barrie J. Carter +8 more
TL;DR: AAV vectors do efficiently promote in vivo gene transfer to the airway epithelium which is stable over several months and indicates that AAV-CFTR vectors could potentially be very useful for gene therapy.
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Mutational Analysis of the Adeno-Associated Virus Type 2 (AAV2) Capsid Gene and Construction of AAV2 Vectors with Altered Tropism
Pei Wu,Wu Xiao,Thomas J. Conlon,Jeffrey A. Hughes,Mavis Agbandje-McKenna,Mavis Agbandje-McKenna,Thomas W. Ferkol,Terence R. Flotte,Nicholas Muzyczka +8 more
TL;DR: In vitro experiments showed that insertion of the serpin receptor ligand in the N-terminal regions of VP1 or VP2 can change the tropism of AAV, and these results provide information on AAV capsid functional domains and are useful for future design of A AV vectors for targeting of specific tissues.
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Defective regulation of outwardly rectifying Cl− channels by protein kinase A corrected by insertion of CFTR
Marie E. Egan,Terence R. Flotte,Terence R. Flotte,Sandra Afione,Rikki Solow,Pamela L. Zeitlin,Barrie J. Carter,William B. Guggino +7 more
TL;DR: It is reported here that expression of recombinant CF genes using adeno-associated virus vectors in CF bronchial epithelial cells corrects defective Cl− secretion, that it induces the appearance of small, linear conductance Cl− channels, and restores protein kinase A activation of outwardly rectifying Cl − channels.