- Fabry disease: current treatment and future perspective
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Yoo HW
- KMID: 2544152
- J Genet Med.
- 2023 Jun;20(1):6-14.
- doi: 10.5734/JGM.2023.20.1.6
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
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Kwak JJ, Kim HR, Byeon SH
- KMID: 2530695
- Yonsei Med J.
- 2022 Jul;63(7):701-705.
- doi: 10.3349/ymj.2022.63.7.701
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
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Choi JW
- KMID: 2515652
- Korean J Otorhinolaryngol-Head Neck Surg.
- 2021 Apr;64(4):215-222.
- doi: 10.3342/kjorl-hns.2020.00836
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
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Paramasivam T, Maiti SK, Palakkara S, R, Mohan D, Manjunthaachar HV, Karthik K, Kumar N
- KMID: 2514870
- Tissue Eng Regen Med.
- 2021 Apr;18(2):235-251.
- doi: 10.1007/s13770-020-00302-3
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
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Kang BK, Jung YT
- KMID: 2512149
- J Bacteriol Virol.
- 2020 Dec;50(4):273-281.
- doi: 10.4167/jbv.2020.50.4.273
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
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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
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Baek SE, Ul-Haq A, Kim DH, Choi HW, Kim MJ, Choi HJ, Kim H
- KMID: 2502473
- Korean J Radiol.
- 2020 Jun;21(6):726-735.
- doi: 10.3348/kjr.2019.0903
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
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Takeoka Y, Yurube T, Nishida K
- KMID: 2501527
- Neurospine.
- 2020 Mar;17(1):3-14.
- doi: 10.14245/ns.2040042.021
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
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Kang YM, Lee SK, Chun YM, Choi YR, Moon SH, Lee HM, Kang HJ
- KMID: 2466340
- Yonsei Med J.
- 2020 Jan;61(1):85-93.
- doi: 10.3349/ymj.2020.61.1.85
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
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Yun IS, Kang E, Ahn HM, Kim YO, Rah DK, Roh TS, Lee WJ, Yun CO
- KMID: 2457481
- Yonsei Med J.
- 2019 Sep;60(9):854-863.
- doi: 10.3349/ymj.2019.60.9.854
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
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Venkatesan JK, Rey-Rico A, Cucchiarini M
- KMID: 2455044
- Tissue Eng Regen Med.
- 2019 Aug;16(4):345-355.
- doi: 10.1007/s13770-019-00179-x
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
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Salkın H, Gönen ZB, Ergen E, Bahar D, Çetin M
- KMID: 2447235
- Int J Stem Cells.
- 2019 Mar;12(1):170-182.
- doi: 10.15283/ijsc18051
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
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Carpena NT, Lee MY
- KMID: 2439210
- Genomics Inform.
- 2018 Dec;16(4):e20.
- doi: 10.5808/GI.2018.16.4.e20
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
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Chung JK, Kim HW, Youn H, Cheon GJ
- KMID: 2422792
- Nucl Med Mol Imaging.
- 2018 Oct;52(5):325-326.
- doi: 10.1007/s13139-018-0540-y
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
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Lukas RV, Wu J, Dey M, Buerki RA, Byrne RW, Dohrmann G
- KMID: 2399593
- J Clin Neurol.
- 2018 Jan;14(1):8-15.
- doi: 10.3988/jcn.2018.14.1.8
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
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Kim YJ
- KMID: 2439836
- Ann Coloproctol.
- 2017 Dec;33(6):207-209.
- doi: 10.3393/ac.2017.33.6.207
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?
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Daliri K, Ljubimov A, Hekmatimoghaddam S
- KMID: 2400866
- Int J Stem Cells.
- 2017 Nov;10(2):119-128.
- doi: 10.15283/ijsc17029
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
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Hwang Y, Kim D, Chang D, Ahn B, Kim YB, Kim G
- KMID: 2412580
- J Vet Sci.
- 2017 Jun;18(2):257-260.
- doi: 10.4142/jvs.2017.18.2.257
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
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Lee MY
- KMID: 2386108
- Korean J Otorhinolaryngol-Head Neck Surg.
- 2017 Jun;60(6):263-270.
- doi: 10.3342/kjorl-hns.2017.00514
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
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Jung BK, Lee WJ, Kang E, Ahn HM, Kim YO, Rah DK, Yun CO, Yun IS
- KMID: 2375215
- Arch Craniofac Surg.
- 2017 Mar;18(1):9-15.
- doi: 10.7181/acfs.2017.18.1.9
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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