Towards a Clinical Trial of Gene Therapy for Retinitis Pigmentosa Associated with Usher Syndrome Type IB

被引：0
|
作者：
Ferla, Rita [1 ,2 ]
Dell'Aquila, Fabio [1 ]
Doria, Monica [1 ]
Trapani, Ivana [1 ]
Ferraiuolo, Maria [3 ]
Noto, Alessia [3 ]
Grazioli, Fabiana [3 ]
Ammendola, Virginia [3 ]
Colloca, Stefano [3 ]
Auricchio, Alberto [1 ]
机构：
[1] Telethon Inst Genet & Med, Pozzuoli, Italy
[2] Univ Naples Federico II, Dept Sci Translat Med, Naples, Italy
[3] Reithera Srl, Castel Romano, Italy
来源：
MOLECULAR THERAPY | 2021年 / 29卷 / 04期
关键词：
D O I：
暂无
中图分类号：
Q81 [生物工程学（生物技术）]; Q93 [微生物学];
学科分类号：
071005 ; 0836 ; 090102 ; 100705 ;
摘要：
255
引用
收藏
页码：131 / 132
页数：2
相关论文
共 50 条
  • [1] Towards a clinical trial of gene therapy for Usher syndrome type IB retinitis pigmentosa
    Dell'Aquila, F.
    Ferla, R.
    Trapani, I.
    Doria, M.
    Ferraiuolo, M.
    Iodice, C.
    Colloca, S.
    Auricchio, A.
    HUMAN GENE THERAPY, 2019, 30 (11) : A108 - A108
  • [2] Towards a clinical trial of gene therapy to target retinitis pigmentosa associated with Usher syndrome type IB
    Ferla, R.
    Dell'Aquila, F.
    Doria, M.
    Ferraiuolo, M.
    Noto, A.
    Grazioli, F.
    Ammendola, V.
    Colloca, S.
    Auricchio, A.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A75 - A75
  • [3] Photodynamic therapy for vasoproliferative tumour associated with retinitis pigmentosa and Usher syndrome type 2
    Grassi, Piergiacomo
    CLINICAL AND EXPERIMENTAL OPTOMETRY, 2022, 105 (01) : 91 - 93
  • [4] Novel RNA-Targeting Gene Therapy Approach for Usher's Syndrome Type II Retinitis Pigmentosa
    Gibbs, Daniel
    Lardelli, Rea
    Nacthrab, Greg
    Roth, Daniela M.
    Lee, Shawn
    Geddes, Claire
    Wilson, Alistair
    Narayan, Nandini
    Zisoulis, Dimitrios
    Batra, Ranjan
    MOLECULAR THERAPY, 2021, 29 (04) : 50 - 50
  • [5] Towards a macaque model for Usher syndrome and autosomal recessive retinitis pigmentosa
    Orten, DJ
    Hemje, AM
    Carney, CA
    Neuringer, M
    Lyons, LA
    Kimberling, WJ
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2003, 44 : U545 - U545
  • [6] PHOSDUCIN AS A CANDIDATE GENE FOR RETINITIS-PIGMENTOSA AND USHER SYNDROME TYPEII
    ARA, F
    JACOBSON, S
    INANA, G
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 1993, 34 (04) : 1458 - 1458
  • [7] A novel gene for Usher syndrome type 2: mutations in the long isoform of whirlin are associated with retinitis pigmentosa and sensorineural hearing loss
    Inga Ebermann
    Hendrik P. N. Scholl
    Peter Charbel Issa
    Elvir Becirovic
    Jürgen Lamprecht
    Bernhard Jurklies
    José M. Millán
    Elena Aller
    Diana Mitter
    Hanno Bolz
    Human Genetics, 2007, 121 : 203 - 211
  • [8] A novel gene for Usher syndrome type 2:: mutations in the long isoform of whirlin are associated with retinitis pigmentosa and sensorineural hearing loss
    Ebermann, Inga
    Scholl, Hendrik P. N.
    Issa, Peter Charbel
    Becirovic, Elvir
    Lamprecht, Juergen
    Jurklies, Bernhard
    Millan, Jose M.
    Aller, Elena
    Mitter, Diana
    Bolz, Hanno
    HUMAN GENETICS, 2007, 121 (02) : 203 - 211
  • [9] Usherin mutations associated with phenotypic variation in Usher syndrome type IIa and retinitis pigmentosa patients.
    Orten, DJ
    Zeigler, T
    Weston, MD
    Carney, CA
    Kimberling, WJ
    INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 2001, 42 (04) : S644 - S644
  • [10] Visual Prognosis in USH2A-Associated Retinitis Pigmentosa Is Worse for Patients with Usher Syndrome Type IIa Than for Those with Nonsyndromic Retinitis Pigmentosa
    Pierrache, Laurence H. M.
    Hartel, Bas P.
    van Wijk, Erwin
    Meester-Smoor, Magda A.
    Cremers, Frans P. M.
    de Baere, Elfride
    de Zaeytijd, Julie
    van Schooneveld, Mary J.
    Cremers, Cor W. R. J.
    Dagnelie, Gislin
    Hoyng, Carel B.
    Bergen, Arthur A.
    Leroy, Bart P.
    Pennings, Ronald J. E.
    van den Born, L. Ingeborgh
    Klaver, Caroline C. W.
    OPHTHALMOLOGY, 2016, 123 (05) : 1151 - 1160
12345