초록

GMP등급 동형접합 백혈구항원형 유도만능줄기세포를 이용하여 관절연골 재생에 최적화된 유리연골 스페로이드를 제작하고, 치료효과를 중대동물에서 무수술적 기법인 MRI를 통하여 효능을 평가함.

(출처 : 요약서 3p)

Abstract ▼

Purpose&Contents
To generate hyaline cartilage using GMP-grade homozygous induced pluripotent stem cells and to confirm its regeneration ability using MRI in large experimental animal models non-invasively.
Production techniques: To establish a technique that can produce hyaline cartilage with

Purpose&Contents
To generate hyaline cartilage using GMP-grade homozygous induced pluripotent stem cells and to confirm its regeneration ability using MRI in large experimental animal models non-invasively.
Production techniques: To establish a technique that can produce hyaline cartilage with high quality using human induced pluripotent stem cells and to confirm its regeneration efficacy through a non-invasive method, MRI.
Evaluation techniques: To set up a proper validity assessment method by constructing an arthritis animal model of middle and large size. To establish sequential and non-invasive MRI evaluation system for accurate determination of cartilage regeneration effect. To confirm the toxicity and oncogenicity ot the generated therapeutics for clinical approval.
Application techniques: To establish SOP documents and perform non-clinical toxicity tests for IND application. To Prepare a clinical trial plan for human induced pluripotent stem cell-derived cartilaginous tissues for transplantation.

Results
1) Establishment of differentiation method of hyaline glass cartilage spheroids using induced pluripotent stem cells
2) Establishment of production and validation methods for hyaline cartilage-specific spheroids
3) Toxicity confirmation of hyaline cartilage spheroids
4) Completion of non-clinical trial evaluation and acquisition of clinical license through medium and large animal models

Expected Contribution
Cartilage is a tissue without regenerative ability, and a regenerative medical treatment strategy is a necessary. Various therapeutic agents have been developed, yet, only the inflammation and pain suppression effects have been confirmed, and the regenerative treatment effects of existing therapeutic agents are still insufficient. If various types of cartilage tissue (especially hyaline cartilage) can be specifically generated into a regeneration drugs, it will be possible to treat degenerative and inflammatory cartilage diseases that have not been treated so far. The development of a cartilage tissue using homozygous human leukocyte antigen-induced pluripotent stem cells has the advantage of minimizing immune rejection as a therapeutic agent considering the possibility of allogeneic transplantation. An injectable cartilage tissue therapeutic therapy will also be used in a wide variety in clinic to increased utilization of regenerative therapy.

(출처 : SUMMARY 5p)

목차 Contents

• 표지 ... 1
• 제 출 문 ... 2
• 보고서 요약서 ... 3
• 국문 요약문 ... 4
• SUMMARY ... 5
• 목차 ... 6
• 1. 연구개발과제의 개요 ... 7
• 1-1. 최종목표 ... 7
• 1-2. 연구개발의 필요성 ... 8
• 1-2. 연구개발의 범위 ... 12
• 2. 국내외 기술개발 현황 ... 15
• 3. 연구수행 내용 및 결과 ... 21
• 4. 목표달성도 및 관련분야 기여도 ... 35
• 4-1. 목표달성도 ... 35
• 5. 연구결과의 활용계획 ... 40
• 6. 연구과정에서 수집한 해외과학기술정보 ... 41
• 7. 연구개발성과의 보안등급 ... 42
• 8. 국가과학기술종합정보시스템에 등록한 연구시설·장비 현황 ... 42
• 9. 연구개발과제 수행에 따른 연구실 등의 안전조치 이행실적 ... 43
• 10. 연구개발과제의 대표적 연구실적 ... 44
• 11. 기타사항 ... 45
• 12. 참고문헌 ... 45
• [별첨4] 첨부11목표제품 특성(TPP; Target Product Profile) ... 46
• [별첨5] TRL기반 연구내용 ... 47
• [별첨4] 실적 증빙자료 ... 48
• 끝페이지 ... 79