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CRISPR technology: A decade of genome editing is only the beginning
JY Wang, JA Doudna - Science, 2023 - science.org
The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …
editing, coupled with advances in computing and imaging capabilities, has initiated a new …
[HTML][HTML] Therapeutic in vivo delivery of gene editing agents
In vivo gene editing therapies offer the potential to treat the root causes of many genetic
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
diseases. Realizing the promise of therapeutic in vivo gene editing requires the ability to …
Strategies for non-viral vectors targeting organs beyond the liver
In recent years, nanoparticles have evolved to a clinical modality to deliver diverse nucleic
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …
acids. Rising interest in nanomedicines comes from proven safety and efficacy profiles …
Advances in oligonucleotide drug delivery
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …
Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors
The development of new CRISPR–Cas genome editing tools continues to drive major
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
advances in the life sciences. Four classes of CRISPR–Cas-derived genome editing agents …
Precise plant genome editing using base editors and prime editors
The development of CRISPR–Cas systems has sparked a genome editing revolution in
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
plant genetics and breeding. These sequence-specific RNA-guided nucleases can induce …
AAV‐mediated gene therapy restores hearing in patients with DFNB9 deafness
J Qi, F Tan, L Zhang, L Lu, S Zhang, Y Zhai… - Advanced …, 2024 - Wiley Online Library
Mutations in OTOFERLIN (OTOF) lead to the autosomal recessive deafness 9 (DFNB9). The
efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is …
efficacy of adeno‐associated virus (AAV)‐mediated OTOF gene replacement therapy is …
The promise and challenge of therapeutic genome editing
JA Doudna - Nature, 2020 - nature.com
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …
cell fates and organism traits, has the potential to both improve our understanding of human …
Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing
CRISPR-Cas9 has emerged as a powerful technology that relies on Cas9/sgRNA
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …
ribonucleoprotein complexes (RNPs) to target and edit DNA. However, many therapeutic …
Biopolymers based aerogels: A review on revolutionary solutions for smart therapeutics delivery
Biopolymers-based aerogels have gained the attention of many scientists in medical and
pharmaceutical applications. Biopolymers-based aerogels are utilized extensively in the …
pharmaceutical applications. Biopolymers-based aerogels are utilized extensively in the …