Targeted drug delivery strategies for precision medicines

MT Manzari, Y Shamay, H Kiguchi, N Rosen… - Nature Reviews …, 2021 - nature.com
Progress in the field of precision medicine has changed the landscape of cancer therapy.
Precision medicine is propelled by technologies that enable molecular profiling, genomic …

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 …

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

M Qiu, Z Glass, J Chen, M Haas, X **… - Proceedings of the …, 2021 - National Acad Sciences
Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are associated with lowered
blood lipid levels, making Angptl3 an attractive therapeutic target for the treatment of human …

[HTML][HTML] Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

T Wei, Q Cheng, YL Min, EN Olson… - Nature communications, 2020 - nature.com
CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …

[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 …

Latest developed strategies to minimize the off-target effects in CRISPR-Cas-mediated genome editing

M Naeem, S Majeed, MZ Hoque, I Ahmad - Cells, 2020 - mdpi.com
Gene editing that makes target gene modification in the genome by deletion or addition has
revolutionized the era of biomedicine. Clustered regularly interspaced short palindromic …

CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy

D Rosenblum, A Gutkin, R Kedmi, S Ramishetti… - Science …, 2020 - science.org
Harnessing CRISPR-Cas9 technology for cancer therapeutics has been hampered by low
editing efficiency in tumors and potential toxicity of existing delivery systems. Here, we …

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 …

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

CA Vakulskas, DP Dever, GR Rettig, R Turk… - Nature medicine, 2018 - nature.com
Translation of the CRISPR–Cas9 system to human therapeutics holds high promise.
However, specificity remains a concern especially when modifying stem cell populations …

Engineered CRISPR–Cas12a variants with increased activities and improved targeting ranges for gene, epigenetic and base editing

BP Kleinstiver, AA Sousa, RT Walton, YE Tak… - Nature …, 2019 - nature.com
Broad use of CRISPR–Cas12a (formerly Cpf1) nucleases has been hindered by the
requirement for an extended TTTV protospacer adjacent motif (PAM). To address this …