Drugging the efferocytosis process: concepts and opportunities

P Mehrotra, KS Ravichandran - Nature reviews Drug discovery, 2022 - nature.com
The daily removal of billions of apoptotic cells in the human body via the process of
efferocytosis is essential for homeostasis. To allow for this continuous efferocytosis, rapid …

Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

MF Dias, K Joo, JA Kemp, SL Fialho… - Progress in retinal and …, 2018 - Elsevier
Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

The TAM family: phosphatidylserine-sensing receptor tyrosine kinases gone awry in cancer

DK Graham, D DeRyckere, KD Davies… - Nature Reviews Cancer, 2014 - nature.com
The TYRO3, AXL (also known as UFO) and MERTK (TAM) family of receptor tyrosine
kinases (RTKs) are aberrantly expressed in multiple haematological and epithelial …

Cellular responses following retinal injuries and therapeutic approaches for neurodegenerative diseases

N Cuenca, L Fernández-Sánchez, L Campello… - Progress in retinal and …, 2014 - Elsevier
Retinal neurodegenerative diseases like age-related macular degeneration, glaucoma,
diabetic retinopathy and retinitis pigmentosa each have a different etiology and …

Manufacturing of recombinant adeno-associated viral vectors for clinical trials

N Clément, JC Grieger - Molecular therapy Methods & clinical development, 2016 - cell.com
The ability to elicit robust and long-term transgene expression in vivo together with minimal
immunogenicity and little to no toxicity are only a few features that make recombinant adeno …

[HTML][HTML] Progress in treating inherited retinal diseases: early subretinal gene therapy clinical trials and candidates for future initiatives

AV Garafalo, AV Cideciyan, E Héon, R Sheplock… - Progress in retinal and …, 2020 - Elsevier
Due to improved phenotyping and genetic characterization, the field of 'incurable'and
'blinding'inherited retinal diseases (IRDs) has moved substantially forward. Decades of …

AAV cis-regulatory sequences are correlated with ocular toxicity

W Xiong, DM Wu, Y Xue, SK Wang… - Proceedings of the …, 2019 - National Acad Sciences
Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …

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 …

Biology and therapy of inherited retinal degenerative disease: insights from mouse models

S Veleri, CH Lazar, B Chang… - Disease models & …, 2015 - journals.biologists.com
Retinal neurodegeneration associated with the dysfunction or death of photoreceptors is a
major cause of incurable vision loss. Tremendous progress has been made over the last two …

Clinical characteristics and current therapies for inherited retinal degenerations

JA Sahel, K Marazova, I Audo - Cold Spring …, 2015 - perspectivesinmedicine.cshlp.org
Inherited retinal degenerations (IRDs) encompass a large group of clinically and genetically
heterogeneous diseases that affect approximately 1 in 3000 people (> 2 million people …