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Nucleic acid drugs: recent progress and future perspectives
X Sun, S Setrerrahmane, C Li, J Hu, H Xu - Signal Transduction and …, 2024 - nature.com
High efficacy, selectivity and cellular targeting of therapeutic agents has been an active area
of investigation for decades. Currently, most clinically approved therapeutics are small …
of investigation for decades. Currently, most clinically approved therapeutics are small …
Splice-modulating antisense oligonucleotides as therapeutics for inherited metabolic diseases
Abstract The last decade (2013–2023) has seen unprecedented successes in the clinical
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
translation of therapeutic antisense oligonucleotides (ASOs). Eight such molecules have …
Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing
Recent advances in drug development have seen numerous successful clinical translations
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …
Targeting the liver with nucleic acid therapeutics for the treatment of systemic diseases of liver origin
A Gogate, J Belcourt, M Shah, AZ Wang… - Pharmacological …, 2024 - Elsevier
Systemic diseases of liver origin (SDLO) are complex diseases in multiple organ systems,
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …
such as cardiovascular, musculoskeletal, endocrine, renal, respiratory, and sensory organ …
The potential of RNA-based therapy for kidney diseases
Inherited kidney diseases (IKDs) are a large group of disorders affecting different nephron
segments, many of which progress towards kidney failure due to the absence of curative …
segments, many of which progress towards kidney failure due to the absence of curative …
Challenges of Assessing Exon 53 Skip** of the Human DMD Transcript with Locked Nucleic Acid-Modified Antisense Oligonucleotides in a Mouse Model for …
Antisense oligonucleotide (AON)-mediated exon skip** is a promising therapeutic
approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression …
approach for Duchenne muscular dystrophy (DMD) patients to restore dystrophin expression …
Next steps for the optimization of exon therapy for Duchenne muscular dystrophy
Introduction It is established that the exon-skip** approach can restore dystrophin in
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …
Duchenne muscular dystrophy (DMD) patients. However, dystrophin restoration levels are …
[HTML][HTML] Oligonucleotide-based therapies for chronic HBV infection: a primer on biochemistry, mechanisms and antiviral effects
A Vaillant - Viruses, 2022 - mdpi.com
Three types of oligonucleotide-based medicines are under clinical development for the
treatment of chronic HBV infection. Antisense oligonucleotides (ASOs) and synthetic …
treatment of chronic HBV infection. Antisense oligonucleotides (ASOs) and synthetic …
[HTML][HTML] Therapeutically significant microRNAs in primary and metastatic brain malignancies
Simple Summary The overall survival of brain cancer patients remains grim, with
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
conventional therapies such as chemotherapy and radiotherapy only providing marginal …
Antisense oligonucleotide-mediated splice switching: potential therapeutic approach for cancer mitigation
Simple Summary Splicing is an important mechanism by which precursor mRNA is modified
into mature mRNA. This splicing plays a major role in the generation of different proteins …
into mature mRNA. This splicing plays a major role in the generation of different proteins …