• Journal of Yeungnam Medical Science
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• 제41권1호
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• pp.13-21
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• 2024

Gene therapy involves the introduction of foreign genetic material into host tissue to alter the expression of genetic products. Gene therapy represents an opportunity to alter the course of various diseases. Hence, genetic products utilizing safe and reliable vectors with improved biotechnology will play a critical role in the treatment of various diseases in the future. This review summarizes various important vectors for gene therapy along with modern techniques for potential craniofacial regeneration using gene therapy. This review also explains current molecular approaches for the management and treatment of cancer using gene therapy. The existing literature was searched to find studies related to gene therapy and its role in craniofacial regeneration and cancer treatment. Various databases such as PubMed, Science Direct, Scopus, Web of Science, and Google Scholar were searched for English language articles using the keywords "gene therapy," "gene therapy in present scenario," "gene therapy in cancer," "gene therapy and vector," "gene therapy in diseases," and "gene therapy and molecular strategies."

• Genomics & Informatics
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• 제16권4호
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• pp.20.1-20.20
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• 2018

Genetic hearing loss crosses almost all the categories of hearing loss which includes the following: conductive, sensory, and neural; syndromic and nonsyndromic; congenital, progressive, and adult onset; high-frequency, low-frequency, or mixed frequency; mild or profound; and recessive, dominant, or sex-linked. Genes play a role in almost half of all cases of hearing loss but effective treatment options are very limited. Genetic hearing loss is considered to be extremely genetically heterogeneous. The advancements in genomics have been instrumental to the identification of more than 6,000 causative variants in more than 150 genes causing hearing loss. Identification of genes for hearing impairment provides an increased insight into the normal development and function of cells in the auditory system. These defective genes will ultimately be important therapeutic targets. However, the auditory system is extremely complex which requires tremendous advances in gene therapy including gene vectors, routes of administration, and therapeutic approaches. This review summarizes and discusses recent advances in elucidating the genomics of genetic hearing loss and technologies aimed at developing a gene therapy that may become a treatment option for in the near future.

• Journal of Interdisciplinary Genomics
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• 제1권1호
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• pp.1-5
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• 2019

Progressive weakness of skeletal muscle is the hallmark of muscular dystrophies. It is often accompanied by cardiomyopathy and respiratory insufficiency. It has generally been perceived as incurable diseases, while the advent of genetic therapy is changing the paradigm. Most research and achievements have been for the treatment of Duchenne muscular dystrophy, while it is promising to hope for therapies for other myopathies. Drugs for nonsense read-through and exon skipping are already approved for clinical use in Europe and the United States, respectively. Gene therapy using adeno-associated virus is in early phase of clinical trial. In this review, most promising genetic therapies will be briefly described.

• 대한물리의학회지
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• 제18권4호
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• pp.29-35
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• 2023

PURPOSE: High-intensity physical therapy may help improve some gross motor developmental delays through an increased treatment frequency. This study describes an increase in physical therapy frequency and intensity over an eight-week time frame for a child with a rare genetic neurodevelopmental disorder. METHODS: A single-subject research was performed. The subject was a nine-year-old boy with MECP2 Duplication Syndrome. The outcome measure consisted of one time before and after the intervention. An intensive physical therapy program was applied to this subject. The treatments included reciprocal crawling, high kneeling, tailor sitting, weight-bearing and shifting training to facilitate ankle balance strategies and training to walk without assistance. RESULTS: After the eight-week follow-up, the child achieved a gradual improvement in the gross motor function measures, and the mother reported that the child frequently engaged in tailor sitting and independently walking. CONCLUSION: This case study highlights that intensive physical therapy improved the overall motor function of a child with MECP2 duplication syndrome.

• 한국동물학회:학술대회논문집
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• 한국동물학회 2001년도 한국생물과학협회 학술발표대회
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• pp.231.1-231
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• 2001

No Abstract, See Full Text

• 한국자원식물학회:학술대회논문집
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• 한국자원식물학회 2002년도 제9차 국제심포지움 및 추계정기학술발표회
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• pp.35-35
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• 2002

• 한국동물학회:학술대회논문집
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• 한국동물학회 1999년도 한국생물과학협회 학술발표대회
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• pp.290.1-290
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• 1999

No Abstract, See Full Text

• BMB Reports
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• 제53권11호
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• pp.565-575
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• 2020

Gene therapy is emerging as a treatment option for inherited genetic diseases. The success of this treatment approach greatly depends upon gene delivery vectors. Researchers have attempted to harness the potential of viral vectors for gene therapy applications over many decades. Among the viral vectors available, gutless adenovirus (GLAd) has been recognized as one of the most promising vectors for in vivo gene delivery. GLAd is constructed by deleting all the viral genes from an adenovirus. Owing to this structural feature, the production of GLAd requires a helper that supplies viral proteins in trans. Conventionally, the helper is an adenovirus. Although the helper adenovirus efficiently provides helper functions, it remains as an unavoidable contaminant and also generates replication-competent adenovirus (RCA) during the production of GLAd. These two undesirable contaminants have raised safety concerns and hindered the clinical applications of GLAd. Recently, we developed helper virus-free gutless adenovirus (HF-GLAd), a new version of GLAd, which is produced by a helper plasmid instead of a helper adenovirus. Utilization of this helper plasmid eliminated the helper adenovirus and RCA contamination in the production of GLAd. HF-GLAd, devoid of helper adenovirus and RCA contaminants, will facilitate its clinical applications. In this review, we discuss the characteristics of adenoviruses, the evolution and production of adenoviral vectors, and the unique features of HF-GLAd as a new platform for gene therapy. Furthermore, we highlight the potential applications of HF-GLAd as a gene delivery vector for the treatment of various inherited genetic diseases.

• Korean Journal of Radiology
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• 제22권11호
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• pp.1858-1874
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• 2021

Recent advances in the molecular and genetic characterization of central nervous system (CNS) tumors have ushered in a new era of tumor classification, diagnosis, and prognostic assessment. In this emerging and rapidly evolving molecular genetic era, imaging plays a critical role in the preoperative diagnosis and surgical planning, molecular marker prediction, targeted treatment planning, and post-therapy assessment of CNS tumors. This review provides an overview of the current imaging methods relevant to the molecular genetic classification of CNS tumors. Specifically, we focused on 1) the correlates between imaging features and specific molecular genetic markers and 2) the post-therapy imaging used for therapeutic assessment.

• Journal of Nutrition and Health
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• 제33권8호
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• pp.813-823
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• 2000

The purpose of this study was to investigate the effects of the nutrition therapy and drug(simvastatin) treatment on the levels of blood lipids for the hyperlipidemic patients according to genetic polymorphism of apo CIII. Subjects of the study consisted of 43(male: 9, female: 34 )hyperlipidemic patients registered to Kyung Hee Medical Center, Intakes of nutrients for the subjects were determined by 24-hr recall method through a personal interviews. The subjects were instructed to take the hypolipidemic and hypocholesterolemic diets. Compliance was monitored through food records, personal interview, and body-weight measurements. The patients changed their basal diet containing 40. 1g fat(182.7mg cholesterol./day) to a diet containing 30.7g fat(139.2mg cholesterol/day) after 12 weeks of nutrition therapy. The distribution of genotypes for apo CIII by Msp I was 32.5% of TT, 50.0% of TC, and 17.5% of CC. aT the beginning of the study, the level of blood triglyceride was the highest for patients with TT and TC. Following the nutrition therapy, patients with CC exhibited the largest drop of blood triglyceride. Following simvastatin treatment with nutrition therapy, blood LDL-cholesterol decreased in most of the patiens with TT. Meanwhile the distribution of genotypes for apo CII by the Fok I was 10.0% of TT, 47.5% of TC, and 42.5% of CC. The level of blood triglyceride was the highest for patients with CC. Following the nutrition therapy, patients with TC exhibited a significant drop for the blood triglyceride. Following simvastatin treatment with nutrition therapy the level of blood LDL-cholesterol decreased in most of the patients with CC. We concluded that dietary habits and food have been changed by nutrition therapy. And blood lipid levels have been decreased by the restriction of intakes of energy, fat and cholesterol. There was variation in the levels of blood lipids according to apo CII polymorphisms. The level of blood lipids responded to nutrition therapy and drug treatment in different was according to genetic polymorphisms. Accordingly, the choice of individualized therapy based on the patient\\`s genetic polymorphism is very important for effective therapy(Korean J Nutrition 33(8) : 813-823, 2000)