Base editing: advances and therapeutic opportunities

EM Porto, AC Komor, IM Slaymaker… - Nature Reviews Drug …, 2020 - nature.com
Base editing—the introduction of single-nucleotide variants (SNVs) into DNA or RNA in
living cells—is one of the most recent advances in the field of genome editing. As around …

[HTML][HTML] Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

S Zhang, J Shen, D Li, Y Cheng - Theranostics, 2021 - ncbi.nlm.nih.gov
CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges

EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …

In vivo somatic cell base editing and prime editing

GA Newby, DR Liu - Molecular Therapy, 2021 - cell.com
Recent advances in genome editing technologies have magnified the prospect of single-
dose cures for many genetic diseases. For most genetic disorders, precise DNA correction is …

Extracellular nanovesicles for packaging of CRISPR-Cas9 protein and sgRNA to induce therapeutic exon skipping

P Gee, MSY Lung, Y Okuzaki, N Sasakawa… - Nature …, 2020 - nature.com
Prolonged expression of the CRISPR-Cas9 nuclease and gRNA from viral vectors may
cause off-target mutagenesis and immunogenicity. Thus, a transient delivery system is …

Engineered Cas9 extracellular vesicles as a novel gene editing tool

X Osteikoetxea, A Silva… - Journal of …, 2022 - Wiley Online Library
Extracellular vesicles (EVs) have shown promise as biological delivery vehicles, but
therapeutic applications require efficient cargo loading. Here, we developed new methods …

Recent advances in CRISPR/Cas9 delivery strategies

BH Yip - Biomolecules, 2020 - mdpi.com
The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system has
revolutionized the field of gene editing. Continuous efforts in developing this technology …

Engineered extracellular vesicles as versatile ribonucleoprotein delivery vehicles for efficient and safe CRISPR genome editing

X Yao, P Lyu, K Yoo, MK Yadav… - Journal of …, 2021 - Wiley Online Library
Transient delivery of CRISPR‐based genome editing effectors is important to reduce off‐
target effects and immune responses. Recently extracellular vesicles (EVs) have been …

Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

S Ling, S Yang, X Hu, D Yin, Y Dai, X Qian… - Nature biomedical …, 2021 - nature.com
Therapeutic genome editing requires effective and targeted delivery methods. The delivery
of Cas9 mRNA using adeno-associated viruses has led to potent in vivo therapeutic efficacy …

[HTML][HTML] TOP-EVs: Technology of Protein delivery through Extracellular Vesicles is a versatile platform for intracellular protein delivery

NF Ilahibaks, AI Ardisasmita, S Xie… - Journal of Controlled …, 2023 - Elsevier
Extracellular vesicles (EVs) have emerged as biocompatible drug delivery vehicles due to
their native ability to deliver bioactive cargo to recipient cells. However, the application of …