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Advancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
Advancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
상세정보
- 자료유형
- 학위논문 서양
- 최종처리일시
- 20250211153125
- ISBN
- 9798346855613
- DDC
- 575
- 서명/저자
- Advancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
- 발행사항
- [Sl] : University of California, Los Angeles, 2024
- 발행사항
- Ann Arbor : ProQuest Dissertations & Theses, 2024
- 형태사항
- 129 p
- 주기사항
- Source: Dissertations Abstracts International, Volume: 86-06, Section: A.
- 주기사항
- Advisor: Kohn, Donald Barry;Lusis, Aldons Jake.
- 학위논문주기
- Thesis (Ph.D.)--University of California, Los Angeles, 2024.
- 초록/해제
- 요약This work encompasses the development of lentiviral technology, and the assessment of viral vector-mediated gene therapy approaches for genetic disorders. Chapter 1 illustrates the development of a high-titer bifunctional lentiviral vector in a vector backbone that has reduced size, high vector yields, and efficient gene transfer to human CD34+ hematopoietic stem and progenitor cells for sickle cell disease (SCD). This lentiviral vector induces high levels of anti-sickling hemoglobins, while concurrently reducing sickle hemoglobin in transduced SCD patient CD34+ cells differentiated into erythrocytes and in the SCD Berkeley mouse model in vivo. Chapter 2 highlights DNA sequencing of peripheral blood cells from Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) patients treated with autologous CD34+ cells transduced with either a γ-retroviral or lentiviral ADA gene vector to assess transgene mutational profiles. We observed reverse transcriptase mutations as well as APOBEC3 guanine (G) to adenosine (A) mutational signatures.
- 일반주제명
- Genetics
- 일반주제명
- Molecular biology
- 일반주제명
- Cellular biology
- 일반주제명
- Music therapy
- 키워드
- Gene therapy
- 키워드
- Viral vectors
- 기타저자
- University of California, Los Angeles Human Genetics 0994
- 기본자료저록
- Dissertations Abstracts International. 86-06A.
- 전자적 위치 및 접속
- 로그인 후 원문을 볼 수 있습니다.
MARC
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■020 ▼a9798346855613
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■040 ▼aMiAaPQ▼cMiAaPQ
■0820 ▼a575
■1001 ▼aHart, Kevyn Lopez.
■24510▼aAdvancements in Viral Vector-Mediated Gene Therapy: Development and Assessment of Lentiviral and γ-Retroviral Approaches for Genetic Hematologic Disorders
■260 ▼a[Sl]▼bUniversity of California, Los Angeles▼c2024
■260 1▼aAnn Arbor▼bProQuest Dissertations & Theses▼c2024
■300 ▼a129 p
■500 ▼aSource: Dissertations Abstracts International, Volume: 86-06, Section: A.
■500 ▼aAdvisor: Kohn, Donald Barry;Lusis, Aldons Jake.
■5021 ▼aThesis (Ph.D.)--University of California, Los Angeles, 2024.
■520 ▼aThis work encompasses the development of lentiviral technology, and the assessment of viral vector-mediated gene therapy approaches for genetic disorders. Chapter 1 illustrates the development of a high-titer bifunctional lentiviral vector in a vector backbone that has reduced size, high vector yields, and efficient gene transfer to human CD34+ hematopoietic stem and progenitor cells for sickle cell disease (SCD). This lentiviral vector induces high levels of anti-sickling hemoglobins, while concurrently reducing sickle hemoglobin in transduced SCD patient CD34+ cells differentiated into erythrocytes and in the SCD Berkeley mouse model in vivo. Chapter 2 highlights DNA sequencing of peripheral blood cells from Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) patients treated with autologous CD34+ cells transduced with either a γ-retroviral or lentiviral ADA gene vector to assess transgene mutational profiles. We observed reverse transcriptase mutations as well as APOBEC3 guanine (G) to adenosine (A) mutational signatures.
■590 ▼aSchool code: 0031.
■650 4▼aGenetics
■650 4▼aMolecular biology
■650 4▼aCellular biology
■650 4▼aMusic therapy
■653 ▼aGene therapy
■653 ▼aLentiviral vectors
■653 ▼aSickle cell disease
■653 ▼aViral vectors
■653 ▼aGenetic hematologic disorders
■690 ▼a0369
■690 ▼a0307
■690 ▼a0379
■690 ▼a0209
■71020▼aUniversity of California, Los Angeles▼bHuman Genetics 0994.
■7730 ▼tDissertations Abstracts International▼g86-06A.
■790 ▼a0031
■791 ▼aPh.D.
■792 ▼a2024
■793 ▼aEnglish
■85640▼uhttp://www.riss.kr/pdu/ddodLink.do?id=T17165116▼nKERIS▼z이 자료의 원문은 한국교육학술정보원에서 제공합니다.
