Scholarly Works - Book sections
Book section
·
January 1, 2020
There are significant engineering challenges in translating gene and nucleic acid delivery from cell and animal models into the clinic. Off-target effects and inefficient delivery to the proper intracellular compartment of the targeted cells are major obst ...
Full text
Cite
Book section
·
January 1, 2019
The recent genome editing revolution has been fueled by the discovery and adaptation of highly specific endonucleases including meganucleases, zinc finger nucleases (ZFNs), TALENs, and CRISPR/Cas9. These genome editing technologies permit user-defined geno ...
Full text
Cite
Book section
·
January 1, 2016
Neuromuscular diseases are a diverse range of conditions that include myopathic and neuropathic disorders related to muscular dysfunction. Inherited neuromuscular diseases are the result of a broad spectrum of genetic mutations, including point mutations, ...
Full text
Cite
Book section
·
January 1, 2015
Modern genome engineering technologies have made the targeted modification of human genes possible for a multitude of therapeutic applications. This chapter discusses the three main platforms that have been developed for targeting specific DNA sequences: z ...
Full text
Cite
Book section
·
January 1, 2010
This chapter describes the potential uses of natural transcriptional regulators, enhancement of these regulators through molecular engineering, and the engineering of entirely synthetic transcription factors for targeted gene regulation. HIF-1 is a heterod ...
Full text
Cite
