scholarly journals Current status of gene therapy and prospect of its clinical application.

1999 ◽  
Vol 22 (6) ◽  
pp. 391-393
Author(s):  
Keiya Ozawa
Keyword(s):  
Gene Therapy ◽  
Clinical Application ◽  
Current Status

Addressing the dark matter of gene therapy: technical and ethical barriers to clinical application

2021 ◽  
Author(s):  
Kateryna Kratzer ◽  
Landon J. Getz ◽  
Thibaut Peterlini ◽  
Jean-Yves Masson ◽  
Graham Dellaire
Keyword(s):  
Gene Therapy ◽  
Dark Matter ◽  
Clinical Application

scholarly journals Current Status of Cardiovascular Gene Therapy

2007 ◽  
Vol 15 (7) ◽  
pp. 1233-1247 ◽  
Author(s):  
Tuomas T Rissanen ◽  
Seppo Ylä-Herttuala
Keyword(s):  
Gene Therapy ◽  
Current Status

Novel Therapeutic Approaches in Sickle Cell Disease

Hematology ◽  
2002 ◽  
Vol 2002 (1) ◽  
pp. 10-34 ◽  
Author(s):  
Mark C. Walters ◽  
Arthur W. Nienhuis ◽  
Elliott Vichinsky
Keyword(s):  
Sickle Cell Disease ◽  
Clinical Application ◽  
Sickle Cell ◽  
Hematopoietic Cell Transplantation ◽  
Pulmonary Complications ◽  
Current Status ◽  
Pulmonary Injury ◽  
Cell Disease ◽  
Therapeutic Approaches ◽  
Novel Treatment

Abstract In this update, selected clinical features of sickle cell disease and their management are reviewed. In addition, the current status of interventions that have curative potential for sickle cell disease is discussed, with particular attention focused on indications, methodology, recent results, and challenges to wider clinical application. In Section I, Dr. Nienhuis describes recent improvements in vector technology, safety, and replacement gene expression that are creating the potential for clinical application of this technology. In Section II, Dr. Vichinsky reviews our current understanding of the pathophysiology and treatment of pulmonary injury in sickle cell disease. The acute and chronic pulmonary complications of sickle cell disease, modulators and predictors of severity, and conventional and novel treatment of these complications are discussed. In Section III, Dr. Walters reviews the current status of hematopoietic cell transplantation for sickle cell disease. Newer efforts to expand its availability by identifying alternate sources of stem cells and by reducing the toxicity of transplantation are discussed.

Abstract 543: Clinical Development of Cardiac Reprogramming Gene Therapy

2020 ◽  
Vol 127 (Suppl_1) ◽  
Author(s):  
Huanyu Zhou ◽  
Laura M Lombardi ◽  
Christopher A Reid ◽  
Jin Yang ◽  
Chetan Srinath ◽  
...  
Keyword(s):  
Heart Failure ◽  
Gene Therapy ◽  
Clinical Application ◽  
Cardiac Fibroblasts ◽  
Target Cells ◽  
Large Animal ◽  
Safety And Efficacy ◽  
Treat Heart Failure ◽  
Ability To Regenerate

Heart failure affects an estimated 38 million people worldwide and is typically caused by cardiomyocyte (CM) loss or dysfunction. Although CMs have limited ability to regenerate, a large pool of non-myocytes, including cardiac fibroblasts (CFs), exist in the postnatal heart. In vivo reprogramming of non-myocytes into functional CMs is emerging as a potential new approach to treat heart failure and substantial proof-of-concept has been achieved in this new field. However, challenges remain in terms of clinical application. First, reported human reprogramming cocktails often consist of five to seven factors that require multiple AAV vectors for delivery. Thus, a less complex cocktail that is able to fit into one AAV vector is needed for this technology to impact human health. Second, the lack of specificity in AAV tropism further complicates the safety and regulatory landscape. A means to limit the expression of reprogramming factors to target cells is critical for maximizing long-term safety. Lastly, although promising studies in small animals have already been reported, safety and efficacy results in large animal MI models are critical to justify cardiac reprogramming in human clinical trials. We have developed a novel human cardiac reprogramming cocktail that consists of only two transcription factors and one miRNA. This new cocktail has been engineered into a single AAV cassette to efficiently reprogram human CFs into cardiomyocytes. We also substantially improved transduction of hCFs through AAV capsid engineering and eliminated CMs expression through a microRNA de-targeting method. Moreover, our novel cardiac reprogramming gene therapy improved cardiac function in both rat and swine MI models upon delivery at various time-points after MI without inducing arrhythmias. Given these promising safety and efficacy results in larger animals, we endeavor to translate direct cardiac reprogramming for clinical application.

scholarly journals Gene Therapy for Liver Cancers: Current Status from Basic to Clinics

Cancers ◽  
2019 ◽  
Vol 11 (12) ◽  
pp. 1865 ◽  
Author(s):  
Kenya Kamimura ◽  
Takeshi Yokoo ◽  
Hiroyuki Abe ◽  
Shuji Terai
Keyword(s):  
Gene Therapy ◽  
Liver Cancer ◽  
Liver Diseases ◽  
Malignant Tumors ◽  
Therapeutic Options ◽  
Endocrine Function ◽  
Current Status ◽  
Congenital Disease ◽  
Gene Therapies ◽  
Liver Cancers

The liver is a key organ for metabolism, protein synthesis, detoxification, and endocrine function, and among liver diseases, including hepatitis, cirrhosis, malignant tumors, and congenital disease, liver cancer is one of the leading causes of cancer-related deaths worldwide. Conventional therapeutic options such as embolization and chemotherapy are not effective against advanced-stage liver cancer; therefore, continuous efforts focus on the development of novel therapeutic options, including molecular targeted agents and gene therapy. In this review, we will summarize the progress toward the development of gene therapies for liver cancer, with an emphasis on recent clinical trials and preclinical studies.

Clinical Application of Bioprosthesis in China: Current Status and Future

2019 ◽  
Vol 39 (4) ◽  
pp. 523-525 ◽  
Author(s):  
Yin Wang ◽  
Wei-wei Jiang ◽  
Nian-guo Dong
Keyword(s):  
Clinical Application ◽  
Current Status

scholarly journals Current Status and Challenges Associated with CNS-Targeted Gene Delivery across the BBB

Pharmaceutics ◽  
2020 ◽  
Vol 12 (12) ◽  
pp. 1216
Author(s):  
Seigo Kimura ◽  
Hideyoshi Harashima
Keyword(s):  
Gene Therapy ◽  
Gene Delivery ◽  
Neurological Disorders ◽  
Viral Vectors ◽  
New Drugs ◽  
Brain Targeting ◽  
Current Status ◽  
Great Promise ◽  
Targeted Gene Delivery ◽  
The Central Nervous System

The era of the aging society has arrived, and this is accompanied by an increase in the absolute numbers of patients with neurological disorders, such as Alzheimer’s disease (AD) and Parkinson’s disease (PD). Such neurological disorders are serious costly diseases that have a significant impact on society, both globally and socially. Gene therapy has great promise for the treatment of neurological disorders, but only a few gene therapy drugs are currently available. Delivery to the brain is the biggest hurdle in developing new drugs for the central nervous system (CNS) diseases and this is especially true in the case of gene delivery. Nanotechnologies such as viral and non-viral vectors allow efficient brain-targeted gene delivery systems to be created. The purpose of this review is to provide a comprehensive review of the current status of the development of successful drug delivery to the CNS for the treatment of CNS-related disorders especially by gene therapy. We mainly address three aspects of this situation: (1) blood-brain barrier (BBB) functions; (2) adeno-associated viral (AAV) vectors, currently the most advanced gene delivery vector; (3) non-viral brain targeting by non-invasive methods.

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