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CRISPR/Cas9 therapeutics: progress and prospects
T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - Signal transduction and …, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …
Drug delivery systems for RNA therapeutics
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …
CRISPR-Cas9 Editing of the HBG1 and HBG2 Promoters to Treat Sickle Cell Disease
Background Sickle cell disease is caused by a defect in the β-globin subunit of adult
hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed …
hemoglobin. Sickle hemoglobin polymerizes under hypoxic conditions, producing deformed …
From target discovery to clinical drug development with human genetics
The substantial investments in human genetics and genomics made over the past three
decades were anticipated to result in many innovative therapies. Here we investigate the …
decades were anticipated to result in many innovative therapies. Here we investigate the …
Sickle cell disease: a review
Importance Sickle cell disease (SCD) is an inherited disorder of hemoglobin, characterized
by formation of long chains of hemoglobin when deoxygenated within capillary beds …
by formation of long chains of hemoglobin when deoxygenated within capillary beds …
Drug delivery systems for CRISPR-based genome editors
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
these drugs must enter the desired cell without eliciting an unwanted immune response, so …
Mechanism and applications of CRISPR/Cas-9-mediated genome editing
Clustered regularly interspaced short palindromic repeat (CRISPR) and their associated
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …
protein (Cas-9) is the most effective, efficient, and accurate method of genome editing tool in …
[HTML][HTML] Biologic and clinical efficacy of LentiGlobin for sickle cell disease
Background Sickle cell disease is characterized by the painful recurrence of vaso-occlusive
events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; …
events. Gene therapy with the use of LentiGlobin for sickle cell disease (bb1111; …
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice
Sickle-cell disease (SCD) is caused by an A· T-to-T· A transversion mutation in the β-globin
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
gene (HBB). Here we show that prime editing can correct the SCD allele (HBB S) to wild …
Gene therapy: Comprehensive overview and therapeutic applications
Gene therapy is the product of man's quest to eliminate diseases. Gene therapy has three
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …
facets namely, gene silencing using siRNA, shRNA and miRNA, gene replacement where …