4.5 Article

First use of gene therapy to treat growth hormone resistant dwarfism in a mouse model

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Review Endocrinology & Metabolism

Laron syndrome - A historical perspective

Zvi Laron et al.

Summary: Laron Syndrome is a rare genetic disorder characterized by dwarfism, obesity, and hypogenitalism. It is caused by deletions or mutations of the GH-receptor gene, with treatment involving the use of recombinant IGF-I. Patients homozygous for GH-R defects are found to be protected from malignancy lifelong, while heterozygotes or double heterozygote subjects are not.

REVIEWS IN ENDOCRINE & METABOLIC DISORDERS (2021)

Review Biotechnology & Applied Microbiology

Current Clinical Applications of In Vivo Gene Therapy with AAVs

Jerry R. Mendell et al.

Summary: Hereditary diseases are caused by gene mutations, affecting millions of Americans, and gene therapy using adeno-associated virus (AAV) has shown promise in treating these diseases. Five treatments have been approved for commercialization, with many more in clinical trials, showcasing the potential of gene therapy in treating a wide range of genetic disorders.

MOLECULAR THERAPY (2021)

Article Biotechnology & Applied Microbiology

A long-term study of AAV gene therapy in dogs with hemophilia A identifies clonal expansions of transduced liver cells

Giang N. Nguyen et al.

Summary: Nine dogs with hemophilia A were treated with AAV gene therapy expressing canine factor VIII and followed for up to 10 years. The therapy corrected the FVIII deficiency in most dogs, with some showing gradual increase in FVIII activity possibly due to clonal expansion of cells. Long-term monitoring for potential genotoxicity is essential for the clinical development of liver-directed AAV gene therapy for hemophilia A.

NATURE BIOTECHNOLOGY (2021)

Article Endocrinology & Metabolism

Effectiveness and safety of rhIGF1 therapy n patients with or without Laron syndrome

Peter Bang et al.

Summary: The study presented data from 242 children and adolescents in 10 European countries receiving rhIGF1 therapy, showing that most treatment-naive/prepubertal children with severe primary IGF1 deficiency were able to promote linear growth, with a safety profile consistent with previous studies.

EUROPEAN JOURNAL OF ENDOCRINOLOGY (2021)

Review Endocrinology & Metabolism

The Actions of IGF-1 in the Growth Plate and Its Role in Postnatal Bone Elongation

Holly L. Racine et al.

CURRENT OSTEOPOROSIS REPORTS (2020)

Review Endocrinology & Metabolism

Tissue-Specific GHR Knockout Mice: An Updated Review

Akash Nagarajan et al.

FRONTIERS IN ENDOCRINOLOGY (2020)

Review Biotechnology & Applied Microbiology

Adeno-associated virus vector as a platform for gene therapy delivery

Dan Wang et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Article Biotechnology & Applied Microbiology

Development of a liver-specific Tet-off AAV8 vector for improved safety of insulin gene therapy for diabetes

Shu Uin Gan et al.

JOURNAL OF GENE MEDICINE (2019)

Article Biochemistry & Molecular Biology

A growth hormone receptor SNP promotes lung cancer by impairment of SOCS2-mediated degradation

Y. Chhabra et al.

ONCOGENE (2018)

Review Endocrinology & Metabolism

IGF-Binding Proteins: Why Do They Exist and Why Are There So Many?

John B. Allard et al.

FRONTIERS IN ENDOCRINOLOGY (2018)

Review Endocrinology & Metabolism

The Growth Hormone Receptor: Mechanism of Receptor Activation, Cell Signaling, and Physiological Aspects

Farhad Dehkhoda et al.

FRONTIERS IN ENDOCRINOLOGY (2018)

Review Oncology

Adeno-Associated Virus (AAV) as a Vector for Gene Therapy

Michael F. Naso et al.

BIODRUGS (2017)

Editorial Material Cell Biology

Epilogue: The future of Laron syndrome - The need for changes

Zvi Laron

GROWTH HORMONE & IGF RESEARCH (2016)

Review Endocrinology & Metabolism

LESSONS FROM 50 YEARS OF STUDY OF LARON SYNDROME

Zvi Laron

ENDOCRINE PRACTICE (2015)

Article Endocrinology & Metabolism

Effectiveness and Safety of rhIGF-1 Therapy in Children: The European Increlex® Growth Forum Database Experience

Peter Bang et al.

HORMONE RESEARCH IN PAEDIATRICS (2015)

Article Biotechnology & Applied Microbiology

Preferential Targeting of Disseminated Liver Tumors Using a Recombinant Adeno-Associated Viral Vector

Marco Della Peruta et al.

HUMAN GENE THERAPY (2015)

Review Pharmacology & Pharmacy

Emerging treatment options for patients with Laron syndrome

Zvi Laron

EXPERT OPINION ON ORPHAN DRUGS (2014)

Article Chemistry, Multidisciplinary

Reversal of experimental Laron Syndrome by xenotransplantation of microencapsulated porcine Sertoli cells

Giovanni Luca et al.

JOURNAL OF CONTROLLED RELEASE (2013)

Article Endocrinology & Metabolism

The Role of GH in Adipose Tissue: Lessons from Adipose-Specific GH Receptor Gene-Disrupted Mice

Edward O. List et al.

MOLECULAR ENDOCRINOLOGY (2013)

Article Endocrinology & Metabolism

The Insulin-Like Growth Factors in Adipogenesis and Obesity

A. Garten et al.

ENDOCRINOLOGY AND METABOLISM CLINICS OF NORTH AMERICA (2012)

Article Biotechnology & Applied Microbiology

Hepatic Gene Transfer in Neonatal Mice by Adeno-Associated Virus Serotype 8 Vector

Lili Wang et al.

HUMAN GENE THERAPY (2012)

Article Multidisciplinary Sciences

The hormonal action of IGF1 in postnatal mouse growth

Elias Stratikopoulos et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Multidisciplinary Sciences

Nuclear targeting of the growth hormone receptor results in dysregulation of cell proliferation and tumorigenesis

Becky L. Conway-Campbell et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2007)

Article Endocrinology & Metabolism

Long-term treatment with recombinant insulin-like growth factor (IGF)-I in children with severe IGF-I deficiency due to growth hormone insensitivity

Steven D. Chernausek et al.

JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM (2007)