Literature DB >> 34580433

Injection pressure levels for creating blebs during subretinal gene therapy.

Brittni A Scruggs1,2, Huber Martins Vasconcelos1,3, Mariana Matioli da Palma1,3, Katie Kogachi1, Mark E Pennesi1, Paul Yang1, Steven T Bailey1, Andreas K Lauer4.   

Abstract

Retinal damage has been associated with increased injection pressure during subretinal gene therapy delivery in various animal models, yet there are no human clinical data regarding the pressures required to initiate and propagate subretinal blebs. This study characterized the intraoperative pressure levels for subretinal gene therapy delivery across eight retinal conditions. A total of 116 patients with retinal degenerative diseases have been treated with subretinal gene therapy at OHSU-Casey Eye Institute as of June 2020; seventy patients (60.3%) were treated using a pneumatic-assisted subretinal delivery system. All retinal blebs were performed using a 41-gauge injection cannula, and use of a balanced salt solution (BSS) "pre-bleb" prior to gene therapy delivery was performed at the discretion of the surgeon. Patient age and intraoperative data for BSS and vector injections were analyzed in a masked fashion for all patients who received pneumatic-assisted subretinal gene therapy. The median age of the patients was 35 years (range 4-70). No significant differences in injection pressures were found across the eight retinal conditions. In this study, patient age was shown to affect maximum injection pressures required for bleb propagation, and the relationship between age and pressure varied based on retinal condition. These data have important implications in optimizing surgical protocols for subretinal injections.
© 2021. The Author(s), under exclusive licence to Springer Nature Limited.

Entities:  

Year:  2021        PMID: 34580433      PMCID: PMC8958181          DOI: 10.1038/s41434-021-00294-2

Source DB:  PubMed          Journal:  Gene Ther        ISSN: 0969-7128            Impact factor:   5.250


  13 in total

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

Authors:  Stephen Russell; Jean Bennett; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Sarah McCague; Dominique Cross; Kathleen A Marshall; Jean Walshire; Taylor L Kehoe; Hannah Reichert; Maria Davis; Leslie Raffini; Lindsey A George; F Parker Hudson; Laura Dingfield; Xiaosong Zhu; Julia A Haller; Elliott H Sohn; Vinit B Mahajan; Wanda Pfeifer; Michelle Weckmann; Chris Johnson; Dina Gewaily; Arlene Drack; Edwin Stone; Katie Wachtel; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2017-07-14       Impact factor: 79.321

2.  Intraoperative Use of Microscope-Integrated Optical Coherence Tomography for Subretinal Gene Therapy Delivery.

Authors:  Ninel Z Gregori; Byron L Lam; Janet L Davis
Journal:  Retina       Date:  2019-10       Impact factor: 4.256

3.  Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.

Authors:  Catherine Cukras; Henry E Wiley; Brett G Jeffrey; H Nida Sen; Amy Turriff; Yong Zeng; Camasamudram Vijayasarathy; Dario Marangoni; Lucia Ziccardi; Sten Kjellstrom; Tae Kwon Park; Suja Hiriyanna; J Fraser Wright; Peter Colosi; Zhijian Wu; Ronald A Bush; Lisa L Wei; Paul A Sieving
Journal:  Mol Ther       Date:  2018-07-07       Impact factor: 11.454

Review 4.  Subretinal Injection: A Review on the Novel Route of Therapeutic Delivery for Vitreoretinal Diseases.

Authors:  Yingqian Peng; Luosheng Tang; Yedi Zhou
Journal:  Ophthalmic Res       Date:  2017-09-01       Impact factor: 2.892

5.  Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia: A Randomized Clinical Trial.

Authors:  M Dominik Fischer; G Alex Ochakovski; Benjamin Beier; Immanuel P Seitz; Yousof Vaheb; Constanze Kortuem; Felix F L Reichel; Laura Kuehlewein; Nadine A Kahle; Tobias Peters; Aniz Girach; Eberhart Zrenner; Marius Ueffing; Robert E MacLaren; Karl Ulrich Bartz-Schmidt; Barbara Wilhelm
Journal:  JAMA Ophthalmol       Date:  2019-11-01       Impact factor: 7.389

6.  Technique of retinal gene therapy: delivery of viral vector into the subretinal space.

Authors:  K Xue; M Groppe; A P Salvetti; R E MacLaren
Journal:  Eye (Lond)       Date:  2017-08-18       Impact factor: 3.775

7.  Optimizing Donor Cellular Dissociation and Subretinal Injection Parameters for Stem Cell-Based Treatments.

Authors:  Brittni A Scruggs; Chunhua Jiao; Cathryn M Cranston; Emily Kaalberg; Kai Wang; Stephen R Russell; Luke A Wiley; Robert F Mullins; Edwin M Stone; Budd A Tucker; Elliott H Sohn
Journal:  Stem Cells Transl Med       Date:  2019-04-19       Impact factor: 6.940

8.  Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

Authors:  Robert E MacLaren; Markus Groppe; Alun R Barnard; Charles L Cottriall; Tanya Tolmachova; Len Seymour; K Reed Clark; Matthew J During; Frans P M Cremers; Graeme C M Black; Andrew J Lotery; Susan M Downes; Andrew R Webster; Miguel C Seabra
Journal:  Lancet       Date:  2014-01-16       Impact factor: 79.321

9.  Intraoperative optical coherence tomographic findings in patients undergoing subretinal gene therapy surgery.

Authors:  Huber M Vasconcelos; Brandon J Lujan; Mark E Pennesi; Paul Yang; Andreas K Lauer
Journal:  Int J Retina Vitreous       Date:  2020-05-01

10.  Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR.

Authors:  Jasmina Cehajic-Kapetanovic; Kanmin Xue; Cristina Martinez-Fernandez de la Camara; Anika Nanda; Alexandra Davies; Laura J Wood; Anna Paola Salvetti; M Dominik Fischer; James W Aylward; Alun R Barnard; Jasleen K Jolly; Edmond Luo; Brandon J Lujan; Tuyen Ong; Aniz Girach; Graeme C M Black; Ninel Z Gregori; Janet L Davis; Potyra R Rosa; Andrew J Lotery; Byron L Lam; Paulo E Stanga; Robert E MacLaren
Journal:  Nat Med       Date:  2020-02-24       Impact factor: 53.440

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