5 research outputs found
AAV Delivery of Wild-Type Rhodopsin Preserves Retinal Function in a Mouse Model of Autosomal Dominant Retinitis Pigmentosa
- Author
- Publication venue
- Mary Ann Liebert, Inc.
- Publication date
- Field of study
Autosomal dominant retinitis pigmentosa (ADRP) is frequently caused by mutations in RHO, the gene for rod photoreceptor opsin. Earlier, a study on mice carrying mutated rhodopsin transgenes on either RHO + / +  or RHO + /– backgrounds suggested that the amount of wild-type rhodopsin affected survival of photoreceptors. Therefore, we treated P23H RHO transgenic mice with adeno-associated virus serotype 5 (AAV5) expressing a cDNA clone of the rhodopsin gene (RHO301) that expressed normal opsin from the mouse opsin promoter. Analysis of the electroretinogram (ERG) demonstrated that increased expression of RHO301 slowed the rate of retinal degeneration in P23H mice: at 6 months, a-wave amplitudes were increased by 100% and b-wave amplitudes by 79%. In contrast, nontransgenic mice injected with AAV5 RHO301 demonstrated a decrease in the ERG, confirming the damaging effect of rhodopsin overproduction in normal photoreceptors. In P23H mice, the increase in the ERG amplitudes was correlated with improvement of retinal structure: the thickness of the outer nuclear layer in RHO301-treated eyes was increased by 80% compared with control eyes. These findings suggest that the wild-type RHO gene can be delivered to rescue retinal degeneration in mice carrying a RHO mutation and that increased production of normal rhodopsin can suppress the effect of the mutated protein. These findings make it possible to treat ADRP caused by different mutations of RHO with the expression of wild-type RHO
Long-Term Rescue of Retinal Structure and Function by Rhodopsin RNA Replacement with a Single Adeno-Associated Viral Vector in P23H RHO
- Author
- Publication venue
- 'Mary Ann Liebert Inc'
- Publication date
- Field of study
Gene Therapy of Inherited Retinopathies: A Long and Successful Road from Viral Vectors to Patients
- Author
- Publication venue
- 'Mary Ann Liebert Inc'
- Publication date
- Field of study
THE ANALYTICAL CHEMISTRY OF THORIUM
- Author
- ABBEY S.
- ABDEL W.
- ABEDINZADEH Z.
- ADAM J.
- AKIMOV V.K.
- AL SHAHRISTANI H.
- ALIMARIN I.P.
- ALIMARIN LP.
- ANTONOV A.V.
- ANTONOV ENKE
- ARCHIMBAUD M.
- ARDEN J.W.
- ARNOLD E.
- ARUSCAVAGE P.J.
- BAEDECKER P.A
- BARKOVSKIL V.F.
- BARNES L L
- BARNES R .
- BARRA F.
- BAZZANO E.
- BERGH F.
- BERGHOLZ J.
- BIMBOT R.
- BLOXAM T.W.
- BOTTEI R S
- BOUWER J.
- BRINKMAN U.A.
- BROOKS R.R.
- BRUNFELT AO.
- BRUNFELT AO.
- BRYCE LT.
- BURMISTROV V.R.
- CALDWELL
- CALDWELL D.L
- CAMERON A.E.
- CAPELIN B.C
- CAPITAN-GARCIA F.
- CERRAI E.
- CHANG F.C
- CHAPMAN J.F.
- CHATTOPADHYAY A.
- CHAUDHARY M.S.
- CHAYLA B.
- CHERRY R.D.
- COHEN N.E.
- CONTARINI M.
- CORRADO T.
- COSPITO M.
- CROSS P.
- CULKIN F.
- CUMMING G.L.
- DAHLBACKA J.
- DALE I.M.
- DAMS R
- DAMS R.
- DANON
- DEAN J .
- DEN BOEF G.
- DERSCHAU VON
- DESAI H.B.
- DIAZ-GUERRA J.P.
- DICKINSON G.W.
- DIEKER J.W.
- DIEKER J.W.
- DIXIT R M
- DONOSCO G.
- DRAGNEV T.
- DRAGULESCU C
- DURHAM R.W.
- EDGINGTON D.N.
- ERDTMANN G.
- EVTIMOVA B.
- FAKHRY A.A.
- FILER T.D.
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- FONTAINE R
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- FUJINAGA T.
- FUNASAKA W„
- FURUKAWA M.
- GALIANO DESANO J.A.
- GALKINA L L
- GANGADHARAM EV.
- GANZERLI-VALENTINI M.T.
- GANZORIG ZH
- GEORGESCU LI.
- GEVORGYAN A M
- GEVORGYAN A.M.
- GIJBELS R.H.
- GLADNEY E.S.
- GLADNEY E.S.
- GOMEZ-PANTOJA
- GOODE G.C
- GORBATYUK O.V.
- GORGENYL T.
- GOTO K.
- GRAY KOWN
- GRISHCHENKO N.V.
- GRITZNER G„ GUTMANN V.
- GUMBAR K.K.
- GUPTA C M
- HANNA Z.G.
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- HERES
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- HSU T.M.
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- ISSA I.M.
- JACOBS J.W.
- JAFFREZIC
- JAIN A K
- JAIN B.
- JAMES G.W.
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- JOHRL K.N.
- KARALOVA Z.K.
- KARALOVA Z.K.
- KATYAL M.
- KAWAHATA M
- KEENAN J.A.
- KEIL
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- KENICHI T.
- KHADEEV V.A
- KHADEEV V.A.
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- KHALIFA H
- KHATER M.M.
- KILEEN P.
- KIRIYAMA T.
- KIRIYAMA T.
- KIRKBRIGHT G.F.
- KIRKBRIGHT G.F.
- KISS T.F.
- KO R.
- KOBASHI A
- KOLESOV G.M.
- KONDRAT'EVA T.M.
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- KOPANICA TARA
- KORCHEMNAYA E.K.
- KORKISCH J.
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- KOTHARL N .
- KRISHNASWAMI S.
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- KUSAKUL P.
- KUZNETSOV V.V.
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- LEIPUNSKAYA D.I.
- LEONI L
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- LOBANOV E.M.
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- MALIK W.U.
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- MARKHABAEV LA.
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- MISHCHENKO V.T.
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- MONSECOUR M.
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- MURATA
- NAKAGAWA G.
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- NEEB K.H.
- NEGOIU D.
- NEJKAR A.D.
- NEMODRUK A.A
- NEVSKAYA E.M.
- NIKOLAEV A.
- NOMURA T„ DONO GAWA
- NORTON E.F.
- OBRUSNIK L
- OEHME F.
- OHNO S.
- OKA Y.
- OKA Y.
- ONISHI H.
- ONISHI KINE
- ORTNER HM.
- OTOMO M.
- OTOSHI IOMI
- PAKALUS P.
- PAKALUS P.
- PALEI P.N.
- PALOMARES J.
- PATEL B.M
- PAVLOVA M.
- PCHELIN V.A.
- PERSIANI C
- PETROW HG.
- PICER M.
- PIESCH E.
- PIRES DE MATOS A
- PLOCK CE.
- PODDAR S.N.
- POLLOCK EN.
- POLYAKOV A I
- POVMEC P.
- PRAKASH S.
- PRASAD S.
- PRASAD S.
- PUCHELT
- PUNG T.C
- PYATOSIN LP.
- RAJKOVIC D.
- RANDA Z.
- RANDA Z.
- RANGACHARI S.
- RAO K .
- REDDY G.R
- REMOV AG.
- ROWE J.J.
- ROWE J.J.
- RUSANOV A.K.
- RYABCHIKOV D.I.
- RYABCHIKOV D.L
- SAKAI T.
- SALBU NNES
- SANCHO J.
- SANDERSON GG.
- SANGAL S.P.
- SAWANT M.
- SCHOCK
- SEVRYUKOV N.N.
- SHARMA C L
- SHARMA C L
- SHARMA C L
- SHARMA G L
- SHARMA V.K.
- SIECK R.F.
- SILL CW.
- SILL CW.
- SINGH M.G.
- SRIVASTAVA H P
- SRIVASTAVA S.P.
- STANTON R.E.
- STEINNES E.
- STEINNES E.
- STEINNES E.
- STUCKLESS J.S.
- STÄRK WSKY
- SUGAWARA M.
- SYROMYATNIKOV N.G.
- SZCZEPANIAK W.
- SZEFERP
- SZYMCZAK S.
- TALVITIE N.A.
- TERIAN R.
- THOMPSON G.M.
- TIKHONOV V.M.
- TRAVESI A
- TSERKOVNITSKAYA LA.
- TURKSTRA J.
- TURKSTRA J· RENS
- TURNHAM D.S.
- UPOR E.
- VAGANOV P.A
- VASILENKO V.D.
- VOLDET P.
- VOLYNETS M.P.
- VYDRA F.
- WAKITA H.
- WEISSBUCH
- WEISZ KISS
- WOLTERMANN H.A.
- YAMAMOTO T.
- ZAITSEV E.L
- ZANELLO P.
- ZHDANOV A.K.
- ZHDANOV AK.
- ZIMMERMAN J.B.
- ZOLOTUKHIN V.K.
- Publication venue
- 'Walter de Gruyter GmbH'
- Publication date
- Field of study
The role of α-synuclein in neurodegeneration — An update
- Author
- A. Abeliovich
- A. Aguzzi
- A. Al-Wandi
- A. Athanassiadou
- A. Beilina
- A. Bellucci
- A. Bellucci
- A. DelleDonne
- A. Elbaz
- A. Esposito
- A. Ferree
- A. Goris
- A. Iwai
- A. Iwata
- A. Mamais
- A. Matsuo
- A. Ohrfelt
- A. Papadimitriou
- A. Pilsl
- A. Raj
- A. Sidhu
- A. Surguchov
- A. Velayati
- A. Weihofen
- A. Zimprich
- A.A. Cooper
- A.B. Knott
- A.B. Manning-Bog
- A.B. Singleton
- A.C. Ferreon
- A.E. Lang
- A.G. Kazantsev
- A.H. Schapira
- A.H. Schapira
- A.H. Tsang
- A.J. Whitworth
- A.J. Williams
- A.K. Berger
- A.K. Lutz
- A.L. Fink
- A.L. Mougenot
- A.M. Cuervo
- A.M. Cuervo
- A.P. Kozikowski
- A.R. Braun
- A.R. Esteves
- A.R. Esteves
- A.R. Esteves
- A.R. Saha
- A.R. Winslow
- A.R. Winslow
- A.S. Hillmer
- A.Y. Abramov
- B. Caughey
- B. Dehay
- B. Dehay
- B. Greten-Harrison
- B. Heeman
- B. Levine
- B. Meeus
- B. Mollenhauer
- B. Mollenhauer
- B. Mollenhauer
- B. Oettinghaus
- B. Spencer
- B. Thomas
- B. Winner
- B. Zheng
- B.C. Campbell
- B.D. Lee
- B.I. Giasson
- B.I. Giasson
- B.I. Giasson
- B.I. Giasson
- B.N. Dugger
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- C. Klein
- C. Lavedan
- C. Linnertz
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- C. Roodveldt
- C. Soto
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- C. Zhou
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- C.B. Lucking
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- C.C. Jao
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- C.H. Hawkes
- C.H. Williams-Gray
- C.L. Kragh
- C.M. Long-Smith
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- D. Sulzer
- D. Trabzuni
- D.A. Amer
- D.A. Scott
- D.D. Murphy
- D.E. Cabin
- D.F. Clayton
- D.G. Healy
- D.J. Moore
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- D.L. Fortin
- D.M. Kay
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- D.M. Maraganore
- D.M. Walsh
- D.P. Karpinar
- D.P. Narendra
- D.W. Dickson
- D.W. Dickson
- D.W. Dickson
- D.W. Miller
- E. Angot
- E. Bezard
- E. Colla
- E. Croisier
- E. Emmanouilidou
- E. Emmanouilidou
- E. Ghebremedhin
- E. Iseki
- E. Israeli
- E. Itakura
- E. Jo
- E. Jo
- E. Junn
- E. Kuusisto
- E. Lindersson
- E. Maries
- E. Masliah
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- E. Sidransky
- E. Sofic
- E.A. Waxman
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- E.A. Waxman
- E.B. Mukaetova-Ladinska
- E.C. Hirsch
- E.H. Norris
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- H.J. Lee
- H.J. Lee
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- H.J. Lee
- H.J. Lee
- H.J. Lee
- H.L. Martin
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- H.Y. Qureshi
- I. Alafuzoff
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- K.A. Jellinger
- K.A. Jellinger
- K.A. Jellinger
- K.A. Jellinger
- K.A. Jellinger
- K.A. Jellinger
- K.A. Jellinger
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- K.F. Winklhofer
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- K.L. Emmer
- K.M. Danzer
- K.M. Danzer
- K.M. Danzer
- K.M. Doyle
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- L. Alvarez-Erviti
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- L. Reiniger
- L. Rojanathammanee
- L. Samaranch
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- M. Filosto
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- M. Goedert
- M. Gu
- M. Hakimi
- M. Hashimoto
- M. Hashimoto
- M. Hashimoto
- M. Ikemura
- M. Inoue
- M. Karbowski
- M. Lebel
- M. Lee
- M. Martinez-Vicente
- M. Martinez-Vicente
- M. Mihajlovic
- M. Murgia
- M. Nagaishi
- M. Neumann
- M. Noguchi-Shinohara
- M. Okochi
- M. Oliveras Salvá
- M. Periquet
- M. Poulopoulos
- M. Riedel
- M. Selikhova
- M. Takahashi
- M. Tanaka
- M. Vila
- M. Xilouri
- M. Xilouri
- M. Xilouri
- M. Zhu
- M. Zhu
- M. Zhu
- M.A. Alim
- M.A. Alim
- M.A. Lynch-Day
- M.A. Nalls
- M.A. Nalls
- M.A. Piggott
- M.B. Aerts
- M.B. Aerts
- M.C. Bennett
- M.C. Bennett
- M.C. Chartier-Harlin
- M.C. Quilty
- M.E. Gegg
- M.E. Gegg
- M.E. Gotz
- M.E. Kalaitzakis
- M.E. Kalaitzakis
- M.F. Beal
- M.F. Chesselet
- M.G. Schlossmacher
- M.G. Schlossmacher
- M.G. Spillantini
- M.G. Spillantini
- M.G. Spillantini
- M.G. Spillantini
- M.G. Spillantini
- M.G. Tansey
- M.H. Polymeropoulos
- M.I. Papp
- M.J. Devine
- M.J. Devine
- M.J. Farrer
- M.J. Park
- M.J. Volles
- M.L. Kramer
- M.L. Kraybill
- M.M. Apetri
- M.M. Wilhelmus
- M.N. Gaugler
- M.N. Sabbagh
- M.R. Cookson
- M.R. Cookson
- M.R. Sierks
- M.S. Forman
- M.T. Lin
- M.W. Dodson
- M.Y. Shino
- N. Badiola
- N. Gosavi
- N. Lev
- N. Ninkina
- N. Ostrerova-Golts
- N. Pankratz
- N. Pankratz
- N. Seto-Salvia
- N. Seto-Salvia
- N. Stefanova
- N. Thayanidhi
- N.B. Cole
- N.M. Bonini
- N.P. Visanji
- N.T. Hettiarachchi
- N.Y. Zhang
- O. Chiba-Falek
- O. Chiba-Falek
- O. Corti
- O. Goker-Alpan
- O. Hornykiewicz
- O. Katsuse
- O. Pletnikova
- O.A. Buneeva
- O.A. Levy
- O.M. El-Agnaf
- O.M. El-Agnaf
- O.M. El-Agnaf
- O.S. Gorbatyuk
- P. Brundin
- P. Brundin
- P. Brundin
- P. Coskun
- P. Davies
- P. Desplats
- P. Garcia-Reitbock
- P. Jenner
- P. Krumova
- P. Lei
- P. Nagley
- P. Pals
- P.E. Spies
- P.G. Foulds
- P.G. Foulds
- P.G. Ince
- P.G. Ince
- P.H. Weinreb
- P.J. Jensen
- P.J. Kahle
- P.J. McLean
- P.J. Spira
- P.K. Auluck
- P.K. Mandal
- P.L. Lantos
- P.L. McGeer
- P.L. McGeer
- P.M. Antony
- P.T. Kotzbauer
- Q. Cai
- Q.L. Ma
- Q.X. Li
- R. Banerjee
- R. Betarbet
- R. Borghi
- R. Bussell Jr.
- R. Cappai
- R. Fernandez-Chacon
- R. Frigerio
- R. Jakes
- R. Kruger
- R. Krüger
- R. Nistico
- R. Sharon
- R. Sharon
- R.A. Fredenburg
- R.A. Moore
- R.E. Burke
- R.G. Perez
- R.H. Kim
- R.H. Swerdlow
- R.J. Perrin
- R.J. Youle
- R.K. Dagda
- R.M. Canet-Aviles
- R.R. Thomas
- R.S. Chaves
- S. Anwar
- S. Arreguin
- S. Chandra
- S. Chandra
- S. DiMauro
- S. Emadi
- S. Engelborghs
- S. Engelender
- S. Fujioka
- S. Gandhi
- S. Geisler
- S. Gilman
- S. Gilman
- S. Greffard
- S. Greffard
- S. Higashi
- S. Jesse
- S. Lesage
- S. Nath
- S. Orimo
- S. Przedborski
- S. Saiki
- S. Saman
- S. Shaikh
- S. Stemberger
- S. Stemberger
- S. Totterdell
- S. Yu
- S.A. Austin
- S.B. Prusiner
- S.E. Winder-Rhodes
- S.F. Falsone
- S.J. Chinta
- S.J. Chung
- S.J. Lee
- S.J. Lee
- S.J. Tabrizi
- S.J. Wood
- S.K. Mak
- S.L. Senior
- S.M. Cardoso
- S.M. Goldman
- S.N. Gomperts
- S.P. Braithwaite
- S.R. Danielson
- S.W. Scholz
- T. Amo
- T. Bartels
- T. Duka
- T. Duka
- T. Gasser
- T. Gasser
- T. Haggerty
- T. Ishizawa
- T. Kanazawa
- T. Kanazawa
- T. Kaul
- T. Lashley
- T. Niki
- T. Nonaka
- T. Ozawa
- T. Ozawa
- T. Pan
- T. Taira
- T. Tokuda
- T. Tokuda
- T. Vogiatzi
- T. Yasuda
- T. Yokota
- T.C. Frank-Cannon
- T.D. Kim
- T.F. Outeiro
- T.G. Beach
- T.G. Beach
- T.J. Montine
- T.L. Yap
- T.M. Dawson
- T.M. Dawson
- V. Askanas
- V. Bogaerts
- V. Bonifati
- V. Choubey
- V. Cullen
- V. Cullen
- V. Deramecourt
- V. Dorval
- V. Licker
- V. Loeb
- V. Munoz-Soriano
- V. Narayanan
- V. Sossi
- V.L. Buchman
- V.M. Lee
- V.M. Nemani
- V.N. Uversky
- V.N. Uversky
- V.N. Uversky
- V.N. Uversky
- V.N. Uversky
- V.N. Uversky
- V.S. Laar Van
- W. Choi
- W. Hoyer
- W. Li
- W. Wang
- W. Westbroek
- W. Xie
- W. Zhang
- W. Zhang
- W. Zhou
- W.J. Schulz-Schaeffer
- W.J. Streit
- W.M. Caudle
- W.P. Gai
- W.P. Gai
- W.R. Galpern
- W.S. Davidson
- W.W. Smith
- W.W. Smith
- X. Chen
- X. Lin
- X. Liu
- X. Peng
- X. Wang
- Y. Arai
- Y. Chu
- Y. Compta
- Y. Compta
- Y. Fan
- Y. Huang
- Y. Li
- Y. Machiya
- Y. Miki
- Y. Peng
- Y. Saito
- Y. Shibasaki
- Y. Tanaka
- Y. Tong
- Y. Usami
- Y. Wang
- Y. Yang
- Y.H. Li
- Y.J. Song
- Y.M. Kim
- Y.S. Kim
- Y.S. Kim
- Y.S. Piao
- Z. Ahmed
- Z. Hong
- Z. Sultana
- Z. Yao
- Publication venue
- 'Walter de Gruyter GmbH'
- Publication date
- Field of study