Morgan L. Maeder

A novel epigenetic editor targeting PCSK9 achieves maximal pharmacological effect with an increased therapeutic window in non-human primates

Frédéric Tremblay, Songlei Liu, Chih‐Wei Ko, Cristiana Giancarlo et autres

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A potent epigenetic editor targeting human PCSK9 for durable reduction of low-density lipoprotein cholesterol levels

Frédéric Tremblay, Qiang Xiong, Chih‐Wei Ko, Kenneth Kelly et autres

Epigenetic editing holds the promise of durable therapeutic effects by silencing disease-causing genes without changing the underlying DNA sequence. In this study, we designed an epigenetic editor to target human PCSK9 and thereby induce DNA methylation at this locus. A single administration …

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An epigenetic editor targeting human PCSK9 efficiently and durably lowers Low Density Lipoprotein Cholesterol (LDL-C) in non-human primates

Francine Tremblay, Kevin R. Kelly, Svati H. Shah, G El Sebae et autres

Abstract Background Reducing the risk of atherosclerotic cardiovascular disease is dependent on both the magnitude and cumulative duration of LDL-C lowering. However, in clinical practice achieving treatment goals is often hampered by low adherence to standard of care. Although PCSK9 inhibitors represent …

Durable Multiplex Epigenetic Editing for Generation of Allogeneic CAR T without Chromosomal Rearrangements

Jamie L. Schafer, Justin D. Trombley, Benjamin Hallisey, Kunza Ahmad et autres

CAR T therapies have provided significant breakthroughs for patients with CD19+ malignancies and multiple myeloma, but a major limitation is the need for individualized manufacturing of autologous T cells, increasing the cost and reducing the availability of these life-saving drugs. An allogeneic …

Exon 13-skipped USH2A protein retains functional integrity in mice, suggesting an exo-skipping therapeutic approach to treat USH2A-associated disease

Nachiket Pendse, Verónica Lamas, Morgan L. Maeder, Basil S. Pawlyk et autres

ABSTRACT Mutations in the USH2A gene are the most common cause of non-syndromic inherited retinal degeneration and Usher syndrome, which is characterized by congenital deafness and progressive vision loss. Development of a vector mediated therapy for USH2A -associated disease has been challenging …

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Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10

Morgan L. Maeder, Michael Stefanidakis, Christopher J. Wilson, Reshica Baral et autres

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In Vivo Assessment of Potential Therapeutic Approaches for USH2A-Associated Diseases

Nachiket Pendse, Verónica Lamas, Basil S. Pawlyk, Morgan L. Maeder et autres

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Somatic Gene Editing of GUCY2D by AAV-CRISPR/Cas9 Alters Retinal Structure and Function in Mouse and Macaque

K. Tyler McCullough, Diego Fajardo, Kaitlyn R. Calabro, Christianne E. Strang et autres

Mutations in GUCY2D , the gene encoding retinal guanylate cyclase-1 (retGC1), are the leading cause of autosomal dominant cone–rod dystrophy (CORD6). Significant progress toward clinical application of gene replacement therapy for Leber congenital amaurosis (LCA) due to recessive mutations in GUCY2D (LCA1) …

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Efficient in vivo editing of CEP290 IVS26 by EDIT-101 as a novel therapeutic for treatment of Leber Congenital Amaurosis 10

Michael Stefanidakis, Morgan L. Maeder, George S. Bounoutas, Clifford Yudkoff et autres

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An AAV-CRISPR/Cas9 gene editing approach for GUCY2D-associated cone rod dystrophy (CORD6)

K. Tyler McCullough, Sanford L. Boye, Diego Fajardo, Christianne E. Strang et autres

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Response to “Unexpected mutations after CRISPR–Cas9 editing in vivo”

Christopher J. Wilson, Tim Fennell, Anne Bothmer, Morgan L. Maeder et autres

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Additional file 9: of UDiTaSâ ˘, a genome editing detection method for indels and genome rearrangements

Georgia Giannoukos, Dawn Ciulla, Eugenio Marco, Hayat Abdulkerim et autres

Figure S8. Comparison of Indel rates between UDiTaS and other methods. T-Cells edited with the TRAC + B2M guides were analyzed for indel editing at the TRAC locus using PCR-amplification followed by Sanger Sequencing or NGS, in addition to UDiTaS with two …