Gene therapy for erythropoietic porphyrias

Cell cycle arrest and p53 prevent ON-target megabase-scale rearrangements induced by CRISPR-Cas9.

Nature communications·2023

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells.

Nature communications·2021

Spleen route accelerates engraftment of human hematopoietic stem cells.

Biochemical and biophysical research communications·2021

Identification of a novel alpha1-antitrypsin variant.

Respiratory medicine case reports·2017

Effect of tyrosine kinase inhibitors on stemness in normal and chronic myeloid leukemia cells.

Leukemia·2016

Hepatoerythropoietic Porphyria Caused by a Novel Homoallelic Mutation in Uroporphyrinogen Decarboxylase Gene in Egyptian Patients.

Folia biologica·2016

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

Gene therapy·2026

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Gene therapy·2026

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Gene therapy·2026

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

Gene therapy·2026

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

Gene therapy·2026

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies.

Gene therapy·2026

Transforming the treatment of autoimmune skin diseases: a journey through biologic therapies.

Expert opinion on biological therapy·2026

Specificity of the Interactions of Folate-Based Vectors with Folate Receptor-α.

The journal of physical chemistry. B·2026

Clinical predictors of early treatment response across systemic therapies in psoriasis: a prospective cohort study.

The Journal of dermatological treatment·2026

Nanobodies targeting SARS-CoV-2 variants.

Acta pharmaceutica Sinica. B·2026

In vivo CRISPR editing for cancer immunotherapy.

Frontiers in immunology·2026

Subcutaneous administration of antibody-drug conjugates: current challenges, emerging opportunities, and future perspectives.

Antibody therapeutics·2026

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Gene therapy for erythropoietic porphyrias

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Cell cycle arrest and p53 prevent ON-target megabase-scale rearrangements induced by CRISPR-Cas9.

CRISPR-Cas9 globin editing can induce megabase-scale copy-neutral losses of heterozygosity in hematopoietic cells.

Spleen route accelerates engraftment of human hematopoietic stem cells.

Identification of a novel alpha1-antitrypsin variant.

Effect of tyrosine kinase inhibitors on stemness in normal and chronic myeloid leukemia cells.

Hepatoerythropoietic Porphyria Caused by a Novel Homoallelic Mutation in Uroporphyrinogen Decarboxylase Gene in Egyptian Patients.

An engineered helper plasmid generates differential E4orf6 and L4-22/33K gene expression increasing AAV vector production.

A gene therapy approach to prevent dilated intercellular space, a hallmark of gastroesophageal reflux disease.

Precision correction of the GJB2 c.235delC mutation by prime editing in vitro.

Bone- and muscle-targeted adeno-associated viral vectors enable tissue-selective vitamin D receptor knockdown in mice.

SACF and GILA for in vitro transformation assessment of CRISPR/Cas9-edited cell therapy candidates: a multi-site study.

rAAV production cost analysis: Indication-specific cost per dose and reduction strategies.

Transforming the treatment of autoimmune skin diseases: a journey through biologic therapies.

Specificity of the Interactions of Folate-Based Vectors with Folate Receptor-α.

Clinical predictors of early treatment response across systemic therapies in psoriasis: a prospective cohort study.

Nanobodies targeting SARS-CoV-2 variants.

In vivo CRISPR editing for cancer immunotherapy.

Subcutaneous administration of antibody-drug conjugates: current challenges, emerging opportunities, and future perspectives.

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Gene therapy for erythropoietic porphyrias

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