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Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis.

Hussain RM, Tran KD, Maguire AM, Berrocal AM.

Ophthalmic Surg Lasers Imaging Retina. 2019 Oct 1;50(10):661-663. doi: 10.3928/23258160-20191009-01.


Safety of Same-Eye Subretinal Sequential Readministration of AAV2-hRPE65v2 in Non-human Primates.

Weed L, Ammar MJ, Zhou S, Wei Z, Serrano LW, Sun J, Lee V, Maguire AM, Bennett J, Aleman TS.

Mol Ther Methods Clin Dev. 2019 Sep 3;15:133-148. doi: 10.1016/j.omtm.2019.08.011. eCollection 2019 Dec 13.


Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.

Yang U, Gentleman S, Gai X, Gorin MB, Borchert MS, Lee TC, Villanueva A, Koenekoop R, Maguire AM, Bennett J, Redmond TM, Nagiel A.

JAMA Ophthalmol. 2019 Oct 3:1-9. doi: 10.1001/jamaophthalmol.2019.3914. [Epub ahead of print]


Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Maguire AM, Russell S, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, Marshall KA, McCague S, Reichert H, Davis M, Simonelli F, Leroy BP, Wright JF, High KA, Bennett J.

Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22.


A History of Trauma is Associated with Aggression, Depression, Non-Suicidal Self-Injury Behavior, and Suicide Ideation in First-Episode Psychosis.

Grattan RE, Lara N, Botello RM, Tryon VL, Maguire AM, Carter CS, Niendam TA.

J Clin Med. 2019 Jul 23;8(7). pii: E1082. doi: 10.3390/jcm8071082.


Visual Function at the Atrophic Border in Choroideremia Assessed with Adaptive Optics Microperimetry.

Tuten WS, Vergilio GK, Young GJ, Bennett J, Maguire AM, Aleman TS, Brainard DH, Morgan JIW.

Ophthalmol Retina. 2019 Oct;3(10):888-899. doi: 10.1016/j.oret.2019.05.002. Epub 2019 May 8.


How physically active do Australian and New Zealander childhood cancer survivors perceive themselves? A report from the ANZCHOG survivorship study.

Mizrahi D, Fardell JE, Wakefield CE, Simar D, Maguire AM, Hubbard G, Cohn RJ; ANZCHOG survivorship study group.

Complement Ther Med. 2019 Jun;44:196-203. doi: 10.1016/j.ctim.2019.04.020. Epub 2019 Apr 25.


Exuberant Anterior Chamber Fibrin Reaction.

Kolomeyer AM, Maguire AM.

Ophthalmol Retina. 2018 May;2(5):440. doi: 10.1016/j.oret.2018.02.010. Epub 2018 May 2. No abstract available.


RDH12 Mutations Cause a Severe Retinal Degeneration With Relatively Spared Rod Function.

Aleman TS, Uyhazi KE, Serrano LW, Vasireddy V, Bowman SJ, Ammar MJ, Pearson DJ, Maguire AM, Bennett J.

Invest Ophthalmol Vis Sci. 2018 Oct 1;59(12):5225-5236. doi: 10.1167/iovs.18-24708.


Variations in the management of acute illness in children with congenital adrenal hyperplasia: An audit of three paediatric hospitals.

Chrisp GL, Maguire AM, Quartararo M, Falhammar H, King BR, Munns CF, Torpy DJ, Hameed S, Rushworth RL.

Clin Endocrinol (Oxf). 2018 Nov;89(5):577-585. doi: 10.1111/cen.13826. Epub 2018 Sep 24.


Salivary cortisol reveals overt and hidden anxiety in survivors of childhood cancer attending clinic.

Amatoury M, Maguire AM, Olivier J, Barton B, Gabriel M, Dalla-Pozza L, Steinbeck KS, Battisti RA.

J Affect Disord. 2018 Nov;240:105-112. doi: 10.1016/j.jad.2018.07.035. Epub 2018 Jul 18.


Identification of a novel pathogenic missense mutation in PRPF31 using whole exome sequencing: a case report.

Bryant L, Lozynska O, Marsh A, Papp TE, van Gorder L, Serrano LW, Gai X, Maguire AM MD, Aleman TS, Bennett J.

Br J Ophthalmol. 2019 Jun;103(6):761-767. doi: 10.1136/bjophthalmol-2017-311405. Epub 2018 Jul 20.


Comparing Clinical Perimetry and Population Receptive Field Measures in Patients with Choroideremia.

Silson EH, Aleman TS, Willett A, Serrano LW, Pearson DJ, Rauschecker AM, Maguire AM, Baker CI, Bennett J, Ashtari M.

Invest Ophthalmol Vis Sci. 2018 Jul 2;59(8):3249-3258. doi: 10.1167/iovs.18-23929.


Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.

Song JY, Aravand P, Nikonov S, Leo L, Lyubarsky A, Bennicelli JL, Pan J, Wei Z, Shpylchak I, Herrera P, Bennett DJ, Commins N, Maguire AM, Pham J, den Hollander AI, Cremers FPM, Koenekoop RK, Roepman R, Nishina P, Zhou S, Pan W, Ying GS, Aleman TS, de Melo J, McNamara I, Sun J, Mills J, Bennett J.

Mol Ther. 2018 Jun 6;26(6):1581-1593. doi: 10.1016/j.ymthe.2018.03.015. Epub 2018 Mar 21.


Attitudes and experiences of childhood cancer survivors transitioning from pediatric care to adult care.

Nandakumar BS, Fardell JE, Wakefield CE, Signorelli C, McLoone JK, Skeen J, Maguire AM, Cohn RJ; ANZCHOG Survivorship Study Group.

Support Care Cancer. 2018 Aug;26(8):2743-2750. doi: 10.1007/s00520-018-4077-5. Epub 2018 Mar 2.


Prescreening whole exome sequencing results from patients with retinal degeneration for variants in genes associated with retinal degeneration.

Bryant L, Lozynska O, Maguire AM, Aleman TS, Bennett J.

Clin Ophthalmol. 2017 Dec 29;12:49-63. doi: 10.2147/OPTH.S147684. eCollection 2018.


On variants and disease-causing mutations: Case studies of a SEMA4A variant identified in inherited blindness.

Bryant L, Lozynska O, Han G, Morgan JIW, Gai X, Maguire AM, Aleman T, Bennett J.

Ophthalmic Genet. 2018 Jan-Feb;39(1):144-146. doi: 10.1080/13816810.2017.1354384. Epub 2017 Aug 14.


Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Russell S, Bennett J, Wellman JA, Chung DC, Yu ZF, Tillman A, Wittes J, Pappas J, Elci O, McCague S, Cross D, Marshall KA, Walshire J, Kehoe TL, Reichert H, Davis M, Raffini L, George LA, Hudson FP, Dingfield L, Zhu X, Haller JA, Sohn EH, Mahajan VB, Pfeifer W, Weckmann M, Johnson C, Gewaily D, Drack A, Stone E, Wachtel K, Simonelli F, Leroy BP, Wright JF, High KA, Maguire AM.

Lancet. 2017 Aug 26;390(10097):849-860. doi: 10.1016/S0140-6736(17)31868-8. Epub 2017 Jul 14. Erratum in: Lancet. 2017 Aug 26;390(10097):848.


Management of Presumed Endogenous Fungal Endophthalmitis in a Child With Acute Lymphoblastic Leukemia.

Uyhazi KE, Kolomeyer AM, Gray IN, Traband A, Kohli AA, O'Brien JM, Maguire AM.

J Pediatr Ophthalmol Strabismus. 2017 Jun 29;54:e42-e46. doi: 10.3928/01913913-20170531-08.


Transition of childhood cancer survivors to adult care: The survivor perspective.

Fardell JE, Wakefield CE, Signorelli C, Hill R, Skeen J, Maguire AM, McLoone JK, Cohn RJ; ANZCHOG survivorship study group.

Pediatr Blood Cancer. 2017 Jun;64(6). doi: 10.1002/pbc.26354. Epub 2016 Nov 5. No abstract available.



Parikh RN, Traband A, Kolomeyer AM, VanderBeek BL, Kim BJ, Maguire AM, Brucker AJ.

Am J Ophthalmol. 2017 May;177:229. doi: 10.1016/j.ajo.2017.02.008. Epub 2017 Mar 18. No abstract available.


Menstrual management in developmentally delayed adolescent females.

Chuah I, McRae A, Matthews K, Maguire AM, Steinbeck K.

Aust N Z J Obstet Gynaecol. 2017 Jun;57(3):346-350. doi: 10.1111/ajo.12595. Epub 2017 Mar 15.


The Role of the Human Visual Cortex in Assessment of the Long-Term Durability of Retinal Gene Therapy in Follow-on RPE65 Clinical Trial Patients.

Ashtari M, Nikonova ES, Marshall KA, Young GJ, Aravand P, Pan W, Ying GS, Willett AE, Mahmoudian M, Maguire AM, Bennett J.

Ophthalmology. 2017 Jun;124(6):873-883. doi: 10.1016/j.ophtha.2017.01.029. Epub 2017 Feb 23.


Intravitreal Bevacizumab for the Treatment of Vitreous Hemorrhage Due to Proliferative Diabetic Retinopathy.

Parikh RN, Traband A, Kolomeyer AM, VanderBeek BL, Kim BJ, Maguire AM, Brucker AJ.

Am J Ophthalmol. 2017 Apr;176:194-202. doi: 10.1016/j.ajo.2017.01.010. Epub 2017 Jan 24.


Natural History of the Central Structural Abnormalities in Choroideremia: A Prospective Cross-Sectional Study.

Aleman TS, Han G, Serrano LW, Fuerst NM, Charlson ES, Pearson DJ, Chung DC, Traband A, Pan W, Ying GS, Bennett J, Maguire AM, Morgan JI.

Ophthalmology. 2017 Mar;124(3):359-373. doi: 10.1016/j.ophtha.2016.10.022. Epub 2016 Dec 13.


Detailed functional and structural phenotype of Bietti crystalline dystrophy associated with mutations in CYP4V2 complicated by choroidal neovascularization.

Fuerst NM, Serrano L, Han G, Morgan JI, Maguire AM, Leroy BP, Kim BJ, Aleman TS.

Ophthalmic Genet. 2016 Dec;37(4):445-452. Epub 2016 Mar 30.


Evaluation of Dose and Safety of AAV7m8 and AAV8BP2 in the Non-Human Primate Retina.

Ramachandran PS, Lee V, Wei Z, Song JY, Casal G, Cronin T, Willett K, Huckfeldt R, Morgan JI, Aleman TS, Maguire AM, Bennett J.

Hum Gene Ther. 2017 Feb;28(2):154-167. doi: 10.1089/hum.2016.111. Epub 2016 Oct 17. Review.


Pneumothorax in neonates: Complication during endotracheal intubation, diagnosis, and management.

Parekh UR, Maguire AM, Emery J, Martin PH.

J Anaesthesiol Clin Pharmacol. 2016 Jul-Sep;32(3):397-9. doi: 10.4103/0970-9185.188820. No abstract available.


Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Bennett J, Wellman J, Marshall KA, McCague S, Ashtari M, DiStefano-Pappas J, Elci OU, Chung DC, Sun J, Wright JF, Cross DR, Aravand P, Cyckowski LL, Bennicelli JL, Mingozzi F, Auricchio A, Pierce EA, Ruggiero J, Leroy BP, Simonelli F, High KA, Maguire AM.

Lancet. 2016 Aug 13;388(10045):661-72. doi: 10.1016/S0140-6736(16)30371-3. Epub 2016 Jun 30.


Primary Gonadal Insufficiency in Male and Female Childhood Cancer Survivors in a Long-Term Follow-Up Clinic.

Gunn HM, Rinne I, Emilsson H, Gabriel M, Maguire AM, Steinbeck KS.

J Adolesc Young Adult Oncol. 2016 Dec;5(4):344-350. Epub 2016 May 19.


Atypical Retinitis in the Setting of Prior Cytomegalovirus Retinitis.

Sandhu HS, Maguire AM.

JAMA Ophthalmol. 2016 Jun 1;134(6):709-10. doi: 10.1001/jamaophthalmol.2015.4986. No abstract available.


Isolated maculopathy associated with biallelic CRB1 mutations.

Shah N, Damani MR, Zhu XS, Bedoukian EC, Bennett J, Maguire AM, Leroy BP.

Ophthalmic Genet. 2017 Mar-Apr;38(2):190-193. doi: 10.3109/13816810.2016.1155225. Epub 2016 Apr 20. No abstract available.


Utility of salivary enzyme immunoassays for measuring estradiol and testosterone in adolescents: a pilot study.

Amatoury M, Lee JW, Maguire AM, Ambler GR, Steinbeck KS.

Int J Adolesc Med Health. 2016 Apr 9;30(1). pii: /j/ijamh.2018.30.issue-1/ijamh-2015-0126/ijamh-2015-0126.xml. doi: 10.1515/ijamh-2015-0126.


Hospital Admission Patterns in Children with CAH: Admission Rates and Adrenal Crises Decline with Age.

Rushworth RL, Falhammar H, Munns CF, Maguire AM, Torpy DJ.

Int J Endocrinol. 2016;2016:5748264. doi: 10.1155/2016/5748264. Epub 2016 Jan 6.


Metabolic Health in Childhood Cancer Survivors: A Longitudinal Study in a Long-Term Follow-Up Clinic.

Gunn HM, Emilsson H, Gabriel M, Maguire AM, Steinbeck KS.

J Adolesc Young Adult Oncol. 2016 Mar;5(1):24-30. doi: 10.1089/jayao.2015.0036. Epub 2015 Dec 8.


Cobalamin C Deficiency Shows a Rapidly Progressing Maculopathy With Severe Photoreceptor and Ganglion Cell Loss.

Bonafede L, Ficicioglu CH, Serrano L, Han G, Morgan JI, Mills MD, Forbes BJ, Davidson SL, Binenbaum G, Kaplan PB, Nichols CW, Verloo P, Leroy BP, Maguire AM, Aleman TS.

Invest Ophthalmol Vis Sci. 2015 Dec;56(13):7875-87. doi: 10.1167/iovs.15-17857.


Evaluation of Ocular Gene Therapy in an Italian Patient Affected by Congenital Leber Amaurosis Type 2 Treated in Both Eyes.

Testa F, Maguire AM, Rossi S, Marshall K, Auricchio A, Melillo P, Bennett J, Simonelli F.

Adv Exp Med Biol. 2016;854:533-9. doi: 10.1007/978-3-319-17121-0_71.


Plasticity of the human visual system after retinal gene therapy in patients with Leber's congenital amaurosis.

Ashtari M, Zhang H, Cook PA, Cyckowski LL, Shindler KS, Marshall KA, Aravand P, Vossough A, Gee JC, Maguire AM, Baker CI, Bennett J.

Sci Transl Med. 2015 Jul 15;7(296):296ra110. doi: 10.1126/scitranslmed.aaa8791.


Discovery of Rare Mutations in Autism: Elucidating Neurodevelopmental Mechanisms.

Gamsiz ED, Sciarra LN, Maguire AM, Pescosolido MF, van Dyck LI, Morrow EM.

Neurotherapeutics. 2015 Jul;12(3):553-71. doi: 10.1007/s13311-015-0363-9. Review.


Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.

Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J.

PLoS One. 2015 Jun 19;10(6):e0129982. doi: 10.1371/journal.pone.0129982. eCollection 2015. No abstract available.


High-resolution adaptive optics retinal imaging of cellular structure in choroideremia.

Morgan JI, Han G, Klinman E, Maguire WM, Chung DC, Maguire AM, Bennett J.

Invest Ophthalmol Vis Sci. 2014 Sep 4;55(10):6381-97. doi: 10.1167/iovs.13-13454.


Late recurrence of retinopathy of prematurity after treatment with both intravitreal bevacizumab and laser.

Chen W, Binenbaum G, Karp K, Baumritter A, Pearson DJ, Maguire AM, Quinn GE.

J AAPOS. 2014 Aug;18(4):402-4. doi: 10.1016/j.jaapos.2014.03.011. Epub 2014 Aug 1.


Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models.

Black A, Vasireddy V, Chung DC, Maguire AM, Gaddameedi R, Tolmachova T, Seabra M, Bennett J.

J Gene Med. 2014 May-Jun;16(5-6):122-30. doi: 10.1002/jgm.2768.


Retinal Structure in Cobalamin C Disease: Mechanistic and Therapeutic Implications.

Aleman TS, Brodie F, Garvin C, Gewaily DY, Ficicioglu CH, Mills MD, Forbes BJ, Maguire AM, Davidson SL.

Ophthalmic Genet. 2015;36(4):339-48. doi: 10.3109/13816810.2014.885059. Epub 2014 Feb 10.


Desmopressin administration in children with central diabetes insipidus: a retrospective review.

Ooi HL, Maguire AM, Ambler GR.

J Pediatr Endocrinol Metab. 2013;26(11-12):1047-52. doi: 10.1515/jpem-2013-0078. Review.


AAV-mediated gene therapy for choroideremia: preclinical studies in personalized models.

Vasireddy V, Mills JA, Gaddameedi R, Basner-Tschakarjan E, Kohnke M, Black AD, Alexandrov K, Zhou S, Maguire AM, Chung DC, Mac H, Sullivan L, Gadue P, Bennicelli JL, French DL, Bennett J.

PLoS One. 2013 May 7;8(5):e61396. doi: 10.1371/journal.pone.0061396. Print 2013. Erratum in: PLoS One. 2015;10(6):e0129982.


Three-year follow-up after unilateral subretinal delivery of adeno-associated virus in patients with Leber congenital Amaurosis type 2.

Testa F, Maguire AM, Rossi S, Pierce EA, Melillo P, Marshall K, Banfi S, Surace EM, Sun J, Acerra C, Wright JF, Wellman J, High KA, Auricchio A, Bennett J, Simonelli F.

Ophthalmology. 2013 Jun;120(6):1283-91. doi: 10.1016/j.ophtha.2012.11.048. Epub 2013 Mar 6.


AAV9 targets cone photoreceptors in the nonhuman primate retina.

Vandenberghe LH, Bell P, Maguire AM, Xiao R, Hopkins TB, Grant R, Bennett J, Wilson JM.

PLoS One. 2013;8(1):e53463. doi: 10.1371/journal.pone.0053463. Epub 2013 Jan 30. Erratum in: PLoS One. 2013;8(8). doi:10.1371/annotation/64b90996-4634-4c63-b737-634397b0b445.


AAV2 gene therapy readministration in three adults with congenital blindness.

Bennett J, Ashtari M, Wellman J, Marshall KA, Cyckowski LL, Chung DC, McCague S, Pierce EA, Chen Y, Bennicelli JL, Zhu X, Ying GS, Sun J, Wright JF, Auricchio A, Simonelli F, Shindler KS, Mingozzi F, High KA, Maguire AM.

Sci Transl Med. 2012 Feb 8;4(120):120ra15. doi: 10.1126/scitranslmed.3002865.


Cerebroretinal microangiopathy with occlusive telangiectasia and multisystemic manifestations.

Choudhry N, Orlin A, Bilaniuk L, Maguire AM.

J AAPOS. 2011 Oct;15(5):502-3. doi: 10.1016/j.jaapos.2011.05.014.


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