Recombinant blood products and therapeutic enzymes: An update

Rajpoot, Kuldeep; Tekade, Muktika; Sharma, Mukesh C.; Sreeharsha, Nagaraja; Sharma, Arti; Tekade, Rakesh K.

doi:10.1016/b978-0-12-814455-8.00013-x

Cited by 1 publication

(1 citation statement)

References 191 publications

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“…To demonstrate that this approach can be generalized to other targets, we chose to upregulate the erythropoietin ( Epo ) gene. The encoded product, Epo, is a 34 kDa serum protein normally secreted by the kidney that regulates erythropoiesis in bone marrow. , Recombinant human Epo protein can be administered as a therapeutic in a variety of clinical scenarios, including chronic renal anemia, HIV infection, and oncologic disorders . Epo is also well-established for the evaluation of in vitro-transcribed mRNA performance, and its physiologic effects of increased red blood cell production can be easily measured from blood samples. ,, …”

Section: Results and Discussionmentioning

confidence: 99%

Robust, Durable Gene Activation In Vivo via mRNA-Encoded Activators

et al. 2022

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Programmable control of gene expression via nuclease-null Cas9 fusion proteins has enabled the engineering of cellular behaviors. Here, both transcriptional and epigenetic gene activation via synthetic mRNA and lipid nanoparticle delivery was demonstrated in vivo. These highly efficient delivery strategies resulted in high levels of activation in multiple tissues. Finally, we demonstrate durable gene activation in vivo via transient delivery of a single dose of a gene activator that combines VP64, p65, and HSF1 with a SWI/SNF chromatin remodeling complex component SS18, representing an important step toward gene-activation-based therapeutics. This induced sustained gene activation could be inhibited via mRNA-encoded AcrIIA4, further improving the safety profile of this approach.

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