000 02802 a2200889 4500
005 20250515192412.0
264 0 _c20091123
008 200911s 0 0 eng d
022 _a1474-547X
024 7 _a10.1016/S0140-6736(09)61836-5
_2doi
040 _aNLM
_beng
_cNLM
100 1 _aMaguire, Albert M
245 0 0 _aAge-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.
_h[electronic resource]
260 _bLancet (London, England)
_cNov 2009
300 _a1597-605 p.
_bdigital
500 _aPublication Type: Clinical Trial, Phase I; Journal Article; Research Support, N.I.H., Extramural; Research Support, Non-U.S. Gov't
650 0 4 _aAdolescent
650 0 4 _aAdult
650 0 4 _aAge Factors
650 0 4 _aBlindness
_xcongenital
650 0 4 _aCarrier Proteins
_xgenetics
650 0 4 _aChild
650 0 4 _aDark Adaptation
650 0 4 _aDependovirus
_xgenetics
650 0 4 _aDisease Progression
650 0 4 _aDose-Response Relationship, Drug
650 0 4 _aElectroretinography
650 0 4 _aEye Proteins
_xgenetics
650 0 4 _aFemale
650 0 4 _aGenetic Therapy
_xmethods
650 0 4 _aGenetic Vectors
_xgenetics
650 0 4 _aHumans
650 0 4 _aInjections
650 0 4 _aMale
650 0 4 _aMutation
_xgenetics
650 0 4 _aNystagmus, Physiologic
650 0 4 _aOptic Atrophy, Hereditary, Leber
_xdiagnosis
650 0 4 _aSafety
650 0 4 _aTreatment Outcome
650 0 4 _aVisual Acuity
650 0 4 _aYoung Adult
650 0 4 _acis-trans-Isomerases
700 1 _aHigh, Katherine A
700 1 _aAuricchio, Alberto
700 1 _aWright, J Fraser
700 1 _aPierce, Eric A
700 1 _aTesta, Francesco
700 1 _aMingozzi, Federico
700 1 _aBennicelli, Jeannette L
700 1 _aYing, Gui-shuang
700 1 _aRossi, Settimio
700 1 _aFulton, Ann
700 1 _aMarshall, Kathleen A
700 1 _aBanfi, Sandro
700 1 _aChung, Daniel C
700 1 _aMorgan, Jessica I W
700 1 _aHauck, Bernd
700 1 _aZelenaia, Olga
700 1 _aZhu, Xiaosong
700 1 _aRaffini, Leslie
700 1 _aCoppieters, Frauke
700 1 _aDe Baere, Elfride
700 1 _aShindler, Kenneth S
700 1 _aVolpe, Nicholas J
700 1 _aSurace, Enrico M
700 1 _aAcerra, Carmela
700 1 _aLyubarsky, Arkady
700 1 _aRedmond, T Michael
700 1 _aStone, Edwin
700 1 _aSun, Junwei
700 1 _aMcDonnell, Jennifer Wellman
700 1 _aLeroy, Bart P
700 1 _aSimonelli, Francesca
700 1 _aBennett, Jean
773 0 _tLancet (London, England)
_gvol. 374
_gno. 9701
_gp. 1597-605
856 4 0 _uhttps://doi.org/10.1016/S0140-6736(09)61836-5
_zAvailable from publisher's website
999 _c19244548
_d19244548