MRNA-based platform based on biology and nanotechnology [Report]



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Delivering an effective therapeutic payload to specific target cells with few adverse effects is considered by many to be the holy grail of medical research. A new Tel Aviv University study explores a biological approach to directing nanocarriers loaded with protein "game changers" to specific cells. The groundbreaking method may be useful in treating myriad malignancies, inflammatory diseases and rare genetic disorders.

Teacher. Dan Peer, Director of the Laboratory of Precision Nanomedicine at the School of Molecular Cell Biology at TAU's Faculty of Life Sciences, led by TAU graduate student Nuphar Veiga and lab colleagues Meir Goldsmith, Yasmin Granot, Daniel Rosenblum, Niels Dammes, Ranit Kedmi and Srinivas Ramishetti. The research was published in Nature Communications.

Over the past few years, lipid carriers encapsulating messenger RNAs (mRNAs) have been shown to be extremely useful in altering the protein expression for a host of diseases. Goal directing this information to specific areas has a major challenge.

"In our new research, we used mRNA-borne nanovehicle carriers carrying a set of genetic instructions via a biological platform called ASSET-to target the genetic instructions of an anti-inflammatory protein in immune cells," says Prof. Peer. "We have been able to demonstrate that selective anti-inflammatory protein in the target cells in reduced symptoms and disease severity in colitis.

"This research is revolutionary. It can be said that it could encode any protein lacking cells, with direct applications for genetic, inflammatory and autoimmune diseases-not to mention cancer, in which certain genes overexpress themselves. "

ASSET (Anchored Secondary scFv Enabling Targeting) uses a biological approach to direct nanocarriers in specific cells to promote gene manipulation.

"This study opens new avenues in cell-specific delivery of mRNA molecules and ultimately provides specific anti-inflammatory (interleukin 10) mRNAs as a novel therapeutic modality for inflammatory bowel diseases," says Ms. Veiga.

"Targeted mRNA-based protein production has both therapeutic and research applications," she concluded. "Going forward, we are interested in using a particular approach for the investigation of novel therapeutics treating inflammation disorders, cancer and rare genetic diseases."

More information:
Nuphar Veiga et al, Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes, Nature Communications (2018). DOI: 10.1038 / s41467-018-06936-1

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Delivering an effective therapeutic payload to specific target cells with few adverse effects is considered by many to be the holy grail of medical research. A new Tel Aviv University study explores a biological approach to directing nanocarriers loaded with protein "game changers" to specific cells. The groundbreaking method may be useful in treating myriad malignancies, inflammatory diseases and rare genetic disorders.

Teacher. Dan Peer, Director of the Laboratory of Precision Nanomedicine at the School of Molecular Cell Biology at TAU's Faculty of Life Sciences, led by TAU graduate student Nuphar Veiga and lab colleagues Meir Goldsmith, Yasmin Granot, Daniel Rosenblum, Niels Dammes, Ranit Kedmi and Srinivas Ramishetti. The research was published in Nature Communications.

Over the past few years, lipid carriers encapsulating messenger RNAs (mRNAs) have been shown to be extremely useful in altering the protein expression for a host of diseases. Goal directing this information to specific areas has a major challenge.

"In our new research, we used mRNA-borne nanovehicle carriers carrying a set of genetic instructions via a biological platform called ASSET-to target the genetic instructions of an anti-inflammatory protein in immune cells," says Prof. Peer. "We have been able to demonstrate that selective anti-inflammatory protein in the target cells in reduced symptoms and disease severity in colitis.

"This research is revolutionary. It can be said that it could encode any protein lacking cells, with direct applications for genetic, inflammatory and autoimmune diseases-not to mention cancer, in which certain genes overexpress themselves. "

ASSET (Anchored Secondary scFv Enabling Targeting) uses a biological approach to direct nanocarriers in specific cells to promote gene manipulation.

"This study opens new avenues in cell-specific delivery of mRNA molecules and ultimately provides specific anti-inflammatory (interleukin 10) mRNAs as a novel therapeutic modality for inflammatory bowel diseases," says Ms. Veiga.

"Targeted mRNA-based protein production has both therapeutic and research applications," she concluded. "Going forward, we are interested in using a particular approach for the investigation of novel therapeutics treating inflammation disorders, cancer and rare genetic diseases."

More information:
Nuphar Veiga et al, Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes, Nature Communications (2018). DOI: 10.1038 / s41467-018-06936-1

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