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[HTML][HTML] Progress in treating inherited retinal diseases: early subretinal gene therapy clinical trials and candidates for future initiatives
Due to improved phenoty** and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …
[HTML][HTML] Gene therapy in retinal dystrophies
Inherited retinal dystrophies (IRDs) are a group of clinically and genetically heterogeneous
degenerative disorders. To date, mutations have been associated with IRDs in over 270 …
degenerative disorders. To date, mutations have been associated with IRDs in over 270 …
Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial
NG Ghazi, EB Abboud, SR Nowilaty, H Alkuraya… - Human genetics, 2016 - Springer
MERTK is an essential component of the signaling network that controls phagocytosis in
retinal pigment epithelium (RPE), the loss of which results in photoreceptor degeneration …
retinal pigment epithelium (RPE), the loss of which results in photoreceptor degeneration …
Leber congenital amaurosis/early-onset severe retinal dystrophy: current management and clinical trials
Leber congenital amaurosis (LCA) is a severe congenital/early-onset retinal dystrophy.
Given its monogenic nature and the immunological and anatomical privileges of the eye …
Given its monogenic nature and the immunological and anatomical privileges of the eye …
[HTML][HTML] Gene Therapy for Retinitis Pigmentosa: Current Challenges and New Progress
Y Liu, X Zong, W Cao, W Zhang, N Zhang, N Yang - Biomolecules, 2024 - mdpi.com
Retinitis pigmentosa (RP) poses a significant threat to eye health worldwide, with
prevalence rates of 1 in 5000 worldwide. This genetically diverse retinopathy is …
prevalence rates of 1 in 5000 worldwide. This genetically diverse retinopathy is …
MERTK mutation update in inherited retinal diseases
I Audo, S Mohand‐Said… - Human …, 2018 - Wiley Online Library
MER tyrosine kinase (MERTK) encodes a surface receptor localized at the apical membrane
of the retinal pigment epithelium. It plays a critical role in photoreceptor outer segment …
of the retinal pigment epithelium. It plays a critical role in photoreceptor outer segment …
Suppression of choroidal neovascularization by AAV-based dual-acting antiangiogenic gene therapy
AL Askou, S Alsing, JNE Benckendorff… - … Therapy Nucleic Acids, 2019 - cell.com
Vascular endothelial growth factor A (VEGFA) is involved in the pathogenesis of
vasoproliferative retinal diseases, such as exudative age-related macular degeneration …
vasoproliferative retinal diseases, such as exudative age-related macular degeneration …
Toward an elucidation of the molecular genetics of inherited retinal degenerations
While individually classed as rare diseases, hereditary retinal degenerations (IRDs) are the
major cause of registered visual handicap in the developed world. Given their hereditary …
major cause of registered visual handicap in the developed world. Given their hereditary …
Long-term characterization of retinal degeneration in Royal College of Surgeons rats using spectral-domain optical coherence tomography
Purpose: Prospective treatments for age-related macular degeneration and inherited retinal
degenerations are commonly evaluated in the Royal College of Surgeons (RCS) rat before …
degenerations are commonly evaluated in the Royal College of Surgeons (RCS) rat before …
Hereditary retinal dystrophy
TC Hohman - Pharmacologic therapy of ocular disease, 2017 - Springer
As our understanding of the genetic basis for inherited retinal disease has expanded, gene
therapy has advanced into clinical development. When the gene mutations associated with …
therapy has advanced into clinical development. When the gene mutations associated with …