In vivo delivery of CRISPR-Cas9 genome editing components for therapeutic applications

K Huang, D Zapata, Y Tang, Y Teng, Y Li - Biomaterials, 2022 - Elsevier
Since its mechanism discovery in 2012 and the first application for mammalian genome
editing in 2013, CRISPR-Cas9 has revolutionized the genome engineering field and created …

Overview of recent advances in nano-based ocular drug delivery

LC Liu, YH Chen, DW Lu - International Journal of Molecular Sciences, 2023 - mdpi.com
Ocular diseases profoundly impact patients' vision and overall quality of life globally.
However, effective ocular drug delivery presents formidable challenges within clinical …

Adeno-associated virus (AAV)-based gene therapies for retinal diseases: where are we?

D Ail, H Malki, EA Zin, D Dalkara - The Application of Clinical …, 2023 - Taylor & Francis
Owing to their small size and safety profiles, adeno-associated viruses (AAVs) have become
the vector of choice for gene therapy applications in the retina. In addition to the naturally …

Unleashing the potential of CRISPR multiplexing: Harnessing Cas12 and Cas13 for precise gene modulation in eye diseases

F Bigini, SH Lee, YJ Sun, Y Sun, VB Mahajan - Vision research, 2023 - Elsevier
Gene therapy is a flourishing field with the potential to revolutionize the treatment of genetic
diseases. The emergence of CRISPR-Cas9 has significantly advanced targeted and efficient …

Biomaterials-mediated CRISPR/Cas9 delivery: recent challenges and opportunities in gene therapy

AK Dubey, E Mostafavi - Frontiers in Chemistry, 2023 - frontiersin.org
The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …

Role of CRISPR/Cas9 in the treatment of Duchenne muscular dystrophy and its delivery strategies

P Agrawal, V Harish, S Mohd, SK Singh, D Tewari… - Life Sciences, 2023 - Elsevier
Duchenne muscular dystrophy (DMD) is a neuromuscular disorder brought on by mutations
in the DMD gene, which prevent muscle cells from expressing the dystrophin protein …

Future perspectives of prime editing for the treatment of inherited retinal diseases

S Hansen, ME McClements, TJ Corydon, RE MacLaren - Cells, 2023 - mdpi.com
Inherited retinal diseases (IRD) are a clinically and genetically heterogenous group of
diseases and a leading cause of blindness in the working-age population. Even though …

[HTML][HTML] Recent advances in carbon monoxide-releasing nanomaterials

X Ning, X Zhu, Y Wang, J Yang - Bioactive Materials, 2024 - Elsevier
As an endogenous signaling molecule, carbon monoxide (CO) has emerged as an
increasingly promising option regarding as gas therapy due to its positive pharmacological …

AAV-based strategies for treatment of retinal and choroidal vascular diseases: advances in age-related macular degeneration and diabetic retinopathy therapies

BFM Castro, JC Steel, CJ Layton - BioDrugs, 2024 - Springer
Age-related macular degeneration (AMD) and diabetic retinopathy (DR) are vascular
diseases with high prevalence, ranking among the leading causes of blindness and vision …

Nanoplatform‐based in vivo gene delivery systems for cancer therapy

R Luo, H Le, Q Wu, C Gong - Small, 2024 - Wiley Online Library
Gene therapy uses modern molecular biology methods to repair disease‐causing genes. As
a burgeoning therapeutic, it has been widely applied for cancer therapy. Since 1989, there …