Amanda M. Dudek
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View article: Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1
Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1 Open
Canonical HIV-1 entry into target cells depends on binding to CD4 as a primary receptor. Because of this, use of the CD4 receptor as a viral trap (a decoy receptor used to prevent infection of target cells) is a promising strategy for the …
View article: Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies
Multilayered HIV-1 resistance in HSPCs through CCR5 Knockout and B cell secretion of HIV-inhibiting antibodies Open
Allogeneic transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment is limited because of the rarity of CCR5-null matched donors, the morbidities assoc…
View article: Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus
Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus Open
In vivo gene therapy targeting hematopoietic stem cells (HSCs) holds significant therapeutic potential for treating hematological diseases. This study uses adeno-associated virus serotype 6 (AAV6) vectors and Cre recombination to sy…
View article: Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus Open
Cystic fibrosis (CF) is a monogenic disease caused by impaired production and/or function of the CF transmembrane conductance regulator (CFTR) protein. Although we have previously shown correction of the most common pathogenic mutation, th…
View article: A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection
A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection Open
View article: Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies
Combining Cell-Intrinsic and -Extrinsic Resistance to HIV-1 By Engineering Hematopoietic Stem Cells for CCR5 Knockout and B Cell Secretion of Therapeutic Antibodies Open
Autologous transplantation of CCR5 null hematopoietic stem and progenitor cells (HSPCs) is the only known cure for HIV-1 infection. However, this treatment is limited because of the rarity of CCR5 -null matched donors, the morbidities asso…
View article: Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells
Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells Open
View article: High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition Open
Therapeutic applications of nuclease-based genome editing would benefit from improved methods for transgene integration via homology-directed repair (HDR). To improve HDR efficiency, we screened six small-molecule inhibitors of DNA-depende…
View article: Transient Inhibition of 53BP1 Increases the Frequency of Targeted Integration in Human Hematopoietic Stem and Progenitor Cells
Transient Inhibition of 53BP1 Increases the Frequency of Targeted Integration in Human Hematopoietic Stem and Progenitor Cells Open
Genome editing by homology directed repair (HDR) is leveraged to precisely modify the genome of therapeutically relevant hematopoietic stem and progenitor cells (HSPCs). Here, we present a new approach to increasing the frequency of HDR in…
View article: A Simultaneous Knock-Out Knock-In Genome Editing Strategy in Hspcs Potently Inhibits Ccr5- and Cxcr4-Tropic Hiv-1 Infection
A Simultaneous Knock-Out Knock-In Genome Editing Strategy in Hspcs Potently Inhibits Ccr5- and Cxcr4-Tropic Hiv-1 Infection Open
View article: Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing
Answered and Unanswered Questions in Early-Stage Viral Vector Transduction Biology and Innate Primary Cell Toxicity for Ex-Vivo Gene Editing Open
Adeno-associated virus is a highly efficient DNA delivery vehicle for genome editing strategies that employ CRISPR/Cas9 and a DNA donor for homology-directed repair. Many groups have used this strategy in development of therapies for blood…
View article: Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus Open
View article: Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus
Targeted replacement of full-length CFTR in human airway stem cells by CRISPR/Cas9 for pan-mutation correction in the endogenous locus Open
Cystic fibrosis (CF) is a monogenic disease caused by impaired production and/or function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein. Although we have previously shown correction of the most common pathogenic…
View article: GPR108 Is a Highly Conserved AAV Entry Factor
GPR108 Is a Highly Conserved AAV Entry Factor Open
View article: Reply to “Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors”
Reply to “Efficient Nuclease-free HR by Clade F AAV Requires High MOIs with High Quality Vectors” Open
We appreciate the interest and comments on our recent report comparing the ability of different adeno-associated virus (AAV) serotypes to mediate targeted genome integration into human CD34+ hematopoietic stem and progenitor cells (HSPCs).…
View article: AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs
AAV6 Is Superior to Clade F AAVs in Stimulating Homologous Recombination-Based Genome Editing in Human HSPCs Open
View article: Differential role for phosphorylation in alternative polyadenylation function versus nuclear import of SR-like protein CPSF6
Differential role for phosphorylation in alternative polyadenylation function versus nuclear import of SR-like protein CPSF6 Open
Cleavage factor I mammalian (CFIm) complex, composed of cleavage and polyadenylation specificity factor 5 (CPSF5) and serine/arginine-like protein CPSF6, regulates alternative polyadenylation (APA). Loss of CFIm function results in proxima…
View article: A Genome-Wide Knock-Out Screen Identifies Novel Host Cell Entry Factor Requirements for Divergent Adeno-Associated Virus Serotypes
A Genome-Wide Knock-Out Screen Identifies Novel Host Cell Entry Factor Requirements for Divergent Adeno-Associated Virus Serotypes Open
Adeno-Associated Virus (AAV) is a non-pathogenic virus that has been harnessed as a vector system for therapeutic gene transfer. Despite decades of research on AAV as a vector, little is known about the molecular determinants required for …
View article: An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor
An Alternate Route for Adeno-associated Virus (AAV) Entry Independent of AAV Receptor Open
Determinants and mechanisms of cell attachment and entry steer adeno-associated virus (AAV) in its utility as a gene therapy vector. Thus far, a systematic assessment of how diverse AAV serotypes engage their proteinaceous receptor AAVR (K…