Improvements in adenoviral vector technology: overcoming barriers for gene therapy

Spinal Muscular Atrophy: More than a Disease of Motor Neurons?

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Expression of the fusogenic p14 FAST protein from a replication-defective adenovirus vector does not provide a therapeutic benefit in an immunocompetent mouse model of cancer.

Cancer gene therapy·2016

Retinal transduction profiles by high-capacity viral vectors.

Gene therapy·2014

At the "junction" of spinal muscular atrophy pathogenesis: the role of neuromuscular junction dysfunction in SMA disease progression.

Current molecular medicine·2013

FSGS-associated alpha-actinin-4 (K256E) impairs cytoskeletal dynamics in podocytes.

Kidney international·2006

Disruption of MAP kinase activation and nuclear factor binding to the IL-12 p40 promoter in HIV-infected myeloid cells.

Clinical and experimental immunology·2004

Rare Novel Genetic Variants of the OFD1 Gene Associated With a Familial Form and a Sporadic Case of Long Bone Atypical Fractures.

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Genetics in Heterotaxy: A Case Series and Literature Review on DNAH9, PKD1L1, MMP21, and GDF1.

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The First Case of Isolated Uterine Atony as the Only Symptom in an Individual With ACTG2-Associated Visceral Myopathy.

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Sotos and Malan Syndromes in Childhood: Molecular and Clinical Findings From a Nationwide Cohort of 48 Patients.

Clinical genetics·2026

Genetic Architecture of Pediatric Cardiomyopathies Assessed by Whole-Exome Sequencing: Insights Into Early-Onset and Syndromic Forms.

Clinical genetics·2026

Two Japanese Cases Highlighting Structural and Phenotypic Overlap in AGO1- and AGO2-Related Neurodevelopmental Disorders.

Clinical genetics·2026

Polycation chain length determines the in vivo mRNA delivery efficiency of block copolymer micelles.

Biomaterials science·2026

Driving Drugs Deeper: Force-Driven Microneedle Systems for Active Therapy and Their Road Towards Clinical Implementation.

Small (Weinheim an der Bergstrasse, Germany)·2026

Mumio Potentiates Temozolomide by Inducing Apoptosis and Ferroptosis in Glioblastoma Cells.

Current pharmaceutical design·2026

Current Targeted Nanoparticle Therapy for Alzheimer's Disease: Current Advances and Future Prospects.

Current neurovascular research·2026

Clinical Presentation, Diagnosis, Treatment, and Outcome of Heart Disease in Becker Muscular Dystrophy.

Anatolian journal of cardiology·2026

Clinical Pharmacology of Eplontersen, the First Approved GalNAc-Conjugated Antisense Oligonucleotide.

Journal of clinical pharmacology·2026

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Spinal Muscular Atrophy: More than a Disease of Motor Neurons?

Expression of the fusogenic p14 FAST protein from a replication-defective adenovirus vector does not provide a therapeutic benefit in an immunocompetent mouse model of cancer.

Retinal transduction profiles by high-capacity viral vectors.

At the "junction" of spinal muscular atrophy pathogenesis: the role of neuromuscular junction dysfunction in SMA disease progression.

FSGS-associated alpha-actinin-4 (K256E) impairs cytoskeletal dynamics in podocytes.

Disruption of MAP kinase activation and nuclear factor binding to the IL-12 p40 promoter in HIV-infected myeloid cells.

Rare Novel Genetic Variants of the OFD1 Gene Associated With a Familial Form and a Sporadic Case of Long Bone Atypical Fractures.

Genetics in Heterotaxy: A Case Series and Literature Review on DNAH9, PKD1L1, MMP21, and GDF1.

The First Case of Isolated Uterine Atony as the Only Symptom in an Individual With ACTG2-Associated Visceral Myopathy.

Sotos and Malan Syndromes in Childhood: Molecular and Clinical Findings From a Nationwide Cohort of 48 Patients.

Genetic Architecture of Pediatric Cardiomyopathies Assessed by Whole-Exome Sequencing: Insights Into Early-Onset and Syndromic Forms.

Two Japanese Cases Highlighting Structural and Phenotypic Overlap in AGO1- and AGO2-Related Neurodevelopmental Disorders.

Polycation chain length determines the in vivo mRNA delivery efficiency of block copolymer micelles.

Driving Drugs Deeper: Force-Driven Microneedle Systems for Active Therapy and Their Road Towards Clinical Implementation.

Mumio Potentiates Temozolomide by Inducing Apoptosis and Ferroptosis in Glioblastoma Cells.

Current Targeted Nanoparticle Therapy for Alzheimer's Disease: Current Advances and Future Prospects.

Clinical Presentation, Diagnosis, Treatment, and Outcome of Heart Disease in Becker Muscular Dystrophy.

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Improvements in adenoviral vector technology: overcoming barriers for gene therapy

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