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Fabry disease: current treatment and future perspective

Yoo HW

Fabry disease (FD), a rare X-linked lysosomal storage disorder, is caused by mutations in the α-galactosidase A gene gene encoding α-galactosidase A (α-Gal A). The functional deficiency of α-Gal A...
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Short-Term Outcomes of the First in Vivo Gene Therapy for RPE65-Mediated Retinitis Pigmentosa

Kwak JJ, Kim HR, Byeon SH

Here, we report early treatment outcomes of gene therapy for early onset retinitis pigmentosa (RP) (Leber congenital amaurosis) associated with biallelic RPE65 mutation in a 30-year-old female patient. Initially, her...
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Inner Ear Gene Therapy in Mouse Models of Genetic Hearing Loss

Choi JW

Hearing loss is the most frequent sensory disorder affecting newborns and children. About 1 newborn in every 500 suffer from congenital hearing loss, with approximately half of these having a...
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Effect of PDGF-B Gene-Activated Acellular Matrix and Mesenchymal Stem Cell Transplantation on Full Thickness Skin Burn Wound in Rat Model

Paramasivam T, Maiti SK, Palakkara S, R, Mohan D, Manjunthaachar HV, Karthik K, Kumar N

BACKGROUND: Full thickness burn wounds are lack of angiogenesis, cell migration, epithelialisation and finally scar tissue formation. Tissue engineered composite graft can provide sustained release of growth factor and promote...
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Semi-Replication-Competent Retroviral Vectors Expressing Gibbon Ape Leukemia Virus Fusogenic Membrane Glycoprotein (GALV FMG) Gene for Cancer Gene Therapy

Kang BK, Jung YT

A semi-replication-competent retroviral (s-RCR) vector system in which the gag-pol and env (GALV FMG, gibbon ape leukemia virus fusogenic membrane glycoprotein) genes were split into two separate packageable vectors was...
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Gene Therapy Options as New Treatment for Inherited Peripheral Neuropathy

Thenmozhi R, Lee JS, Park NY, Choi BO, Hong YB

  • KMID: 2503979
  • Exp Neurobiol.
  • 2020 Jun;29(3):177-188.
  • doi: 10.5607/en20004
Inherited peripheral neuropathy (IPN) is caused by heterogeneous genetic mutations in more than 100 genes. So far, several treatment options for IPN have been developed and clinically evaluated using small...
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Human Organic Anion Transporting Polypeptide 1B3 Applied as an MRI-Based Reporter Gene

Baek SE, Ul-Haq A, Kim DH, Choi HW, Kim MJ, Choi HJ, Kim H

Objective: Recent innovations in biology are boosting gene and cell therapy, but monitoring the response to these treatments is difficult. The purpose of this study was to find an MRI-reporter...
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Gene Therapy Approach for Intervertebral Disc Degeneration: An Update

Takeoka Y, Yurube T, Nishida K

Intervertebral disc degeneration is the primary cause of back pain and associated with neurological disorders including radiculopathy, myelopathy, and paralysis. The currently available surgical treatments predominantly include the excision of...
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Follistatin Mitigates Myofibroblast Differentiation and Collagen Synthesis of Fibroblasts from Scar Tissue around Injured Flexor Tendons

Kang YM, Lee SK, Chun YM, Choi YR, Moon SH, Lee HM, Kang HJ

PURPOSE: The aim of this study was to investigate the effect of FST gene on the inhibition of fibrosis in fibroblastic cells from scar tissue around repaired zone II flexor...
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Effect of Relaxin Expression from an Alginate Gel-Encapsulated Adenovirus on Scar Remodeling in a Pig Model

Yun IS, Kang E, Ahn HM, Kim YO, Rah DK, Roh TS, Lee WJ, Yun CO

PURPOSE: Relaxin (RLX) is a transforming growth factor-β1 (TGF-β1) antagonist that is believed to function as a potent collagen re-arranger and a major suppressor of extracellular matrix components. Adenoviruses (Ads)...
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Current Trends in Viral Gene Therapy for Human Orthopaedic Regenerative Medicine

Venkatesan JK, Rey-Rico A, Cucchiarini M

BACKGROUND: Viral vector-based therapeutic gene therapy is a potent strategy to enhance the intrinsic reparative abilities of human orthopaedic tissues. However, clinical application of viral gene transfer remains hindered by...
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Effects of TGF-β1 Overexpression on Biological Characteristics of Human Dental Pulp-derived Mesenchymal Stromal Cells

Salkın H, Gönen ZB, Ergen E, Bahar D, Çetin M

OBJECTIVE: The aim of our study was to investigate the effect of Transforming growth factor beta-1 (TGF-β1) gene therapy on the surface markers, multilineage differentiation, viability, apoptosis, cell cycle, DNA...
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Genetic Hearing Loss and Gene Therapy

Carpena NT, Lee MY

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...
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Sodium Iodide Symporter (NIS) in the Management of Patients with Thyroid Carcinoma

Chung JK, Kim HW, Youn H, Cheon GJ

Although radioiodine has been applied in thyroid diseases including carcinoma for over 70 years, it was only in 1996 that the basic molecular mechanism of iodine uptake was identified. Iodide...
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A Survey of the Neuro-Oncology Landscape

Lukas RV, Wu J, Dey M, Buerki RA, Byrne RW, Dohrmann G

The field of neuro-oncology is evolving rapidly. Many important advances have recently been reported, and other promising investigations have the potential to soon make substantial impacts in the field, especially...
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The Future Medical Science and Colorectal Surgeons

Kim YJ

Future medical technology breakthroughs will build from the incredible progress made in computers, biotechnology, and nanotechnology and from the information learned from the human genome. With such technology and information,...
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Glaucoma, Stem Cells, and Gene Therapy: Where Are We Now?

Daliri K, Ljubimov A, Hekmatimoghaddam S

Glaucoma is the second most common cause of blindness, affecting 70~80 million people around the world. The death of retinal ganglion cells (RGCs) is the main cause of blindness related...
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Effects of neural stem cells and 5-fluorocytosine in canine metastatic lung tumor

Hwang Y, Kim D, Chang D, Ahn B, Kim YB, Kim G

This is the first case report to describe the tumor regressive effect of systemic human neural stem cell (NSC)/5-fluorocytosine (5-FC) therapy on canine metastatic lung tumor. The therapeutic effects appeared...
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Gene Therapy for Hearing Protection

Lee MY

Sensorineural hearing loss (SNHL) does not recover and only few exceptions exist. It is mostly due to the reason that hair cells in the cochlea cannot regenerate once damaged. Therefore,...
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Effect of Relaxin Expressing Adenovirus on Scar Remodeling: A Preliminary Study

Jung BK, Lee WJ, Kang E, Ahn HM, Kim YO, Rah DK, Yun CO, Yun IS

BACKGROUND: Relaxin is a transforming growth factor β1 antagonist. To determine the effects of relaxin on scar reduction, we investigated the scar remodeling process by injecting relaxin-expressing adenoviruses using a...
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