Search Results - "Doerfler, Alexandria M."

A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing by Li, Ang, Lee, Ciaran M, Hurley, Ayrea E, Jarrett, Kelsey E, De Giorgi, Marco, Lu, Weiqi, Balderrama, Karol S, Doerfler, Alexandria M, Deshmukh, Harshavardhan, Ray, Anirban, Bao, Gang, Lagor, William R

“…Adeno-associated viral (AAV) vectors packaging the CRISPR-Cas9 system (AAV-CRISPR) can efficiently modify disease-relevant genes in somatic tissues with high…”
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Depletion of essential isoprenoids and ER stress induction following acute liver-specific deletion of HMG-CoA reductase by De Giorgi, Marco, Jarrett, Kelsey E., Burton, Jason C., Doerfler, Alexandria M., Hurley, Ayrea, Li, Ang, Hsu, Rachel H., Furgurson, Mia, Patel, Kalyani R., Han, Jun, Borchers, Christoph H., Lagor, William R.

“…HMG-CoA reductase (Hmgcr) is the rate-limiting enzyme in the mevalonate pathway and is inhibited by statins. In addition to cholesterol, Hmgcr activity is also…”
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LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study by Doerfler, Alexandria M., Park, So Hyun, Assini, Julia M., Youssef, Amer, Saxena, Lavanya, Yaseen, Adam B., De Giorgi, Marco, Chuecos, Marcel, Hurley, Ayrea E., Li, Ang, Marcovina, Santica M., Bao, Gang, Boffa, Michael B., Koschinsky, Marlys L., Lagor, William R.

“…Lipoprotein(a) (Lp(a)) represents a unique subclass of circulating lipoprotein particles and consists of an apolipoprotein(a) (apo(a)) molecule covalently…”
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Targeting the Apoa1 locus for liver-directed gene therapy by De Giorgi, Marco, Li, Ang, Hurley, Ayrea, Barzi, Mercedes, Doerfler, Alexandria M., Cherayil, Nikitha A., Smith, Harrison E., Brown, Jonathan D., Lin, Charles Y., Bissig, Karl-Dimiter, Bao, Gang, Lagor, William R.

“…Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless…”
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AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9 by Li, Ang, Tanner, Mark R., Lee, Ciaran M., Hurley, Ayrea E., De Giorgi, Marco, Jarrett, Kelsey E., Davis, Timothy H., Doerfler, Alexandria M., Bao, Gang, Beeton, Christine, Lagor, William R.

“…Adeno-associated viral (AAV) vectors are a leading candidate for the delivery of CRISPR-Cas9 for therapeutic genome editing in vivo. However, AAV-based…”
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Somatic Editing of Ldlr With Adeno-Associated Viral-CRISPR Is an Efficient Tool for Atherosclerosis Research by Jarrett, Kelsey E, Lee, Ciaran, De Giorgi, Marco, Hurley, Ayrea, Gillard, Baiba K, Doerfler, Alexandria M, Li, Ang, Pownall, Henry J, Bao, Gang, Lagor, William R

“…OBJECTIVE—Atherosclerosis studies in Ldlr knockout mice require breeding to homozygosity and congenic status on C57BL6/J background, a process that is both…”
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High levels of lipoprotein(a) in transgenic mice exacerbate atherosclerosis and promote vulnerable plaque features in a sex-specific manner by Assini, Julia M., Clark, Justin R., Youssef, Amer, Xing, Chuce, Doerfler, Alexandria M., Park, So Hyun, Saxena, Lavanya, Yaseen, Adam B., Børen, Jan, Gros, Robert, Bao, Gang, Lagor, William R., Boffa, Michael B., Koschinsky, Marlys L.

“…Despite increased clinical interest in lipoprotein(a) (Lp(a)), many questions remain about the molecular mechanisms by which it contributes to atherosclerotic…”
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Recent advances in genome editing for cardiovascular disease by Doerfler, Alexandria M., Walkey, Christopher J., Lagor, William R.

“…PURPOSE OF REVIEWThis review highlights recent progress in applying genome editing to the study and treatment of cardiovascular disease (CVD). RECENT…”
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Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment by Doerfler, Alexandria M., Han, Jun, Jarrett, Kelsey E., Tang, Li, Jain, Antrix, Saltzman, Alexander, De Giorgi, Marco, Chuecos, Marcel, Hurley, Ayrea E., Li, Ang, Morand, Pauline, Ayala, Claudia, Goodlett, David R., Malovannaya, Anna, Martin, James F., de Aguiar Vallim, Thomas Q., Shroyer, Noah, Lagor, William R.

“…The intestine occupies the critical interface between cholesterol absorption and excretion. Surprisingly little is known about the role of de novo cholesterol…”
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Abstract 425: Genetic Inhibition Of Hepatic Dolichol Biosynthesis Results In Hypolipidemia by De Giorgi, Marco, Doerfler, Alexandria M, Hurley, Ayrea, Walkey, Christopher, Lagor, William R

“…Dolichol is a nonsterol isoprenoid derived from the mevalonate pathway. Dehydrodolichyl diphosphate synthase subunit (DHDDS) is a branch point enzyme that - in…”
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Abstract 123: Intestinal Deletion Of 3-hydroxy-3-methylglutaryl-coenzyme A Reductase Promotes Expansion Of The Resident Stem Cell Compartment by Doerfler, Alexandria M, Han, Jun, Tang, Li, De Giorgi, Marco, Jarrett, Kelsey E, Hurley, Ayrea, Li, Ang, Chuecos, Marcel, Morand, Pauline, Ayala, Claudia, Martin, James F, Goodlett, David R, Vallim, Thomas A, Shroyer, Noah, Lagor, William R

“…Abstract only While the intestine is the critical interface between cholesterol absorption and excretion, surprisingly little is known about the role of de…”
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Abstract 216: Modeling Statin Hepatotoxicity with Acute Liver Specific Deletion of HmgCoA Reductase by De Giorgi, Marco, Jarrett, Kelsey E, Burton, Jason C, Doerfler, Alexandria M, Hurley, Ayrea, Hsu, Rachel H, Lagor, William R

“…Abstract only Hmg-CoA Reductase (Hmgcr) catalyzes the conversion of Hmg-CoA to mevalonate, which is the rate-limiting step in the cholesterol biosynthetic…”
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Intestinal Deletion of 3-Hydroxy-3-Methylglutaryl-Coenzyme A Reductase Promotes Expansion of the Resident Stem Cell Compartment by Doerfler, Alexandria M., Han, Jun, Jarrett, Kelsey E., Tang, Li, Jain, Antrix, Saltzman, Alexander, De Giorgi, Marco, Chuecos, Marcel, Hurley, Ayrea E., Li, Ang, Morand, Pauline, Ayala, Claudia, Goodlett, David R., Malovannaya, Anna, Martin, James F., de Aguiar Vallim, Thomas Q., Shroyer, Noah, Lagor, William R.

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Somatic editing of Ldlr with AAV-CRISPR is an efficient tool for atherosclerosis research by Jarrett, Kelsey E., Lee, Ciaran, De Giorgi, Marco, Hurley, Ayrea, Gillard, Baiba K., Doerfler, Alexandria M., Li, Ang, Pownall, Henry J., Bao, Gang, Lagor, William R.

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Abstract 032: Somatic Editing of Ldlr with AAV-CRISPR for Atherosclerosis Studies by Jarrett, Kelsey E, Doerfler, Alexandria M, Hurley, Ayrea, Hsu, Rachel H, De Giorgi, Marco, Li, Ang, Pownall, Henry J, Lee, Ciaran, Bao, Gang, Lagor, William R

“…Abstract only Background: Current methods to study genetic contributions to atherosclerosis are time-consuming and expensive. The Ldlr and Apoe knockout (KO)…”
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Depletion of essential isoprenoids and ER stress induction following acute liver-specific deletion of HMG-CoA reductase by De Giorgi, Marco, Jarrett, Kelsey E, Burton, Jason C, Doerfler, Alexandria M, Hurley, Ayrea, Li, Ang, Hsu, Rachel H, Furgurson, Mia, Patel, Kalyani R, Han, Jun, Borchers, Christoph H, Lagor, William R

“…HMG-CoA reductase (Hmgcr) is the rate-limiting enzyme in the mevalonate pathway and is inhibited by statins. In addition to cholesterol, Hmgcr activity is also…”
Get full text

Depletion of essential isoprenoids and ER stress induction following acute liver-specific deletion of HMG-CoA reductase by De Giorgi, Marco, Jarrett, Kelsey E, Burton, Jason C, Doerfler, Alexandria M, Hurley, Ayrea, Li, Ang, Hsu, Rachel H, Furgurson, Mia, Patel, Kalyani R, Han, Jun, Borchers, Christoph H, Lagor, William R

“…HMG-CoA reductase (Hmgcr) is the rate-limiting enzyme in the mevalonate pathway and is inhibited by statins. In addition to cholesterol, Hmgcr activity is also…”
Get full text