Design of Lipid and Polymeric Carriers for Nucleic Acid Delivery

Zhu, Lin

doi:10.21007/etd.cghs.2010.0384

Cited by 1 publication

(1 citation statement)

References 212 publications

(277 reference statements)

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“…95 Lipid and polymer toxicity is often alleviated or minimized by the use of biodegradable formulations with readily degraded ester or amide bonds. 96,97 While these formulations have been successful at limiting some of the associated toxicity, there is much work to be done to ensure safe and effective delivery in healthy subjects, including the synthesis and screening of biodegradable polymers and lipids with an even safer toxicity profile. The advent of new targeting modalities, such as the small peptide targeting groups discovered using in vivo phage display 98 or the use of lipids with tropisms for specific tissues and organs, coupled with biodegradable polymers and lipids, can increase specificity while decreasing toxicity.…”

Section: ■ Translation To Clinical Applicationsmentioning

confidence: 99%

Genome Editing: Insights from Chemical Biology to Support Safe and Transformative Therapeutic Applications

et al. 2017

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Programmable nuclease-based genome editing technologies, including the clustered, regularly interspaced, short palindromic repeats (CRISPR)/Cas9 system, are becoming an essential component of many applications ranging from agriculture to medicine. However, fundamental limitations currently prevent the widespread, safe, and practical use of genome editors, especially for human disease interventions. These limitations include off-target effects, a lack of control over editing activity, suboptimal DNA repair outcomes, insufficient target conversion, and inadequate delivery performance. This perspective focuses on the potential for biological chemistry to address these limitations such that newly developed genome editing technologies can enable the broadest range of potential future applications. Equally important will be the development of these powerful technologies within a relevant ethical framework that emphasizes safety and responsible innovation.

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