Marianna Romito
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View article: Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR
Unveiling the cut-and-repair cycle of designer nucleases in human stem and T cells via CLEAR-time dPCR Open
DNA repair mechanisms in human primary cells, including error-free repair, and, recurrent nuclease cleavage events, remain largely uncharacterised. We elucidate gene-editing related repair processes using Cleavage and Lesion Evaluation via…
View article: MEGA dPCR reveals chromosomal aberrations, NHEJ precise repair, recurrent nuclease cleavage and DSB half-life
MEGA dPCR reveals chromosomal aberrations, NHEJ precise repair, recurrent nuclease cleavage and DSB half-life Open
Advances in designer DNA editors, such as programmable nucleases, PASTE, Prime, and base editors, have recently proved their therapeutic value and potential to treat genetic disorders. However, a comprehensive understanding of their cellul…
View article: CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells Open
Interleukin 7 Receptor alpha Severe Combined Immunodeficiency (IL7R-SCID) is a life-threatening disorder caused by homozygous mutations in the IL7RA gene. Defective IL7R expression in humans hampers T cell precursors' proliferation …
View article: CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells
CRISPR/Cas9-based disease modelling and functional correction of Interleukin 7 Receptor alpha Severe Combined Immunodeficiency in T-lymphocytes and hematopoietic stem cells Open
Interleukin 7 Receptor α Severe Combined Immunodeficiency (IL7R-SCID) is a life-threatening disorder caused by homozygous mutations in the IL7RA gene. Defective IL7R expression in humans hampers T cell precursors proliferation and differen…
View article: Preclinical Evaluation of a Novel TALEN Targeting <i>CCR5</i> Confirms Efficacy and Safety in Conferring Resistance to HIV‐1 Infection
Preclinical Evaluation of a Novel TALEN Targeting <i>CCR5</i> Confirms Efficacy and Safety in Conferring Resistance to HIV‐1 Infection Open
Therapies to treat patients infected with human immunodeficiency virus (HIV) aim at preventing viral replication but fail to eliminate the virus. Although transplantation of allogeneic CCR5 Δ32 homozygous stem cell grafts provided a cure f…
View article: Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome
Targeted gene correction of human hematopoietic stem cells for the treatment of Wiskott - Aldrich Syndrome Open
View article: Clinically relevant CCR5 editing in primary CD4+ T cells with TALEN confers resistance to HIV-1 infection
Clinically relevant CCR5 editing in primary CD4+ T cells with TALEN confers resistance to HIV-1 infection Open
Today’s available therapies to treat patients infected with human immunodeficiency virus (HIV) aim at preventing viral replication and transmission but fail to eliminate the virus. Although transplantation of an allogeneic CCR5Δ32 homozygo…
View article: Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells
Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells Open
View article: Genome editing for blood disorders: state of the art and recent advances
Genome editing for blood disorders: state of the art and recent advances Open
In recent years, tremendous advances have been made in the use of gene editing to precisely engineer the genome. This technology relies on the activity of a wide range of nuclease platforms — such as zinc-finger nucleases, transcription ac…
View article: Designer epigenome modifiers enable robust and sustained gene silencing in clinically relevant human cells
Designer epigenome modifiers enable robust and sustained gene silencing in clinically relevant human cells Open
Targeted modulation of gene expression represents a valuable approach to understand the mechanisms governing gene regulation. In a therapeutic context, it can be exploited to selectively modify the aberrant expression of a disease-causing …
View article: Failure to detect DNA-guided genome editing using Natronobacterium gregoryi Argonaute
Failure to detect DNA-guided genome editing using Natronobacterium gregoryi Argonaute Open