成人霊長類の赤緑色失明に対する遺伝子治療
Katherine Mancuso1, William W Hauswirth, Qiuhong Li
1Department of Ophthalmology, Box 356485, University of Washington, 1959 North East Pacific Street, Seattle, Washington 98195, USA.
Nature
|September 18, 2009
まとめ
遺伝子療法は,色盲の成人の猿の三色色彩視を成功裏に回復させました. この画期的な発見は,大人の先天性視力障害の治療は可能であり,色盲の治療への希望を示しています.
科学分野:
- オフタルモロジック (眼科)
- 遺伝学 遺伝学とは
- 神経科学は神経科学である.
背景:
- 赤緑色盲症は,欠落しているLまたはM光色素によって引き起こされる一般的な遺伝疾患です.
- 生まれながらの視力障害は,発達過程の初期に依存しているため,成人では治療できないと考えられていた.
研究 の 目的:
- 成人霊長類における赤緑色失明を治すための遺伝子療法の可能性を調査する.
- 新しいコーン・ピグメントが成人期にトリクロマティック視力を回復できるかどうかを判断する.
主な方法:
- 遺伝子療法は,成人色盲類猿の網膜に第3のオプシンを導入した.
- 行動テストは,三色色彩視力の回復を評価した.
主要な成果:
- 3つ目のコーン・ピグメントの添加により,成人の被験者にトリクロマティック・カラービジョンを可能にしました.
- 早期の発達過程を必要とせずに,色の視力の回復が成功しました.
結論:
- 遺伝子療法は,先天性色盲の成人の霊長類で三色色彩視を確立することができます.
- 成人の先天性視力障害の治療は実行可能であり,以前の発達上の制約に挑戦します.
関連する概念動画
Green Algae
756
Green algae, also referred to as chlorophytes, are different from red algae in having the chloroplasts containing chlorophylls a and b, which give them their distinct green hue. However, they lack phycobiliproteins, preventing them from developing the red or blue-green pigmentation seen in red algae. In terms of photosynthetic pigment composition, green algae closely resemble plants and share a close evolutionary relationship with them. Taxonomically Green algae belong to Phylum Chlorophyta in...
756
Red Algae
796
Red algae, also known as rhodophytes, are primarily found in marine environments, though some species inhabit freshwater and terrestrial ecosystems. These organisms exist in both unicellular and multicellular forms, with some multicellular varieties reaching macroscopic sizes.As phototrophic organisms, red algae contain chlorophyll a; however, their chloroplasts lack chlorophyll b. Instead, they possess phycobiliproteins, which serve as major light-harvesting pigments, similar to those found in...
796
Gene Therapy
27.4K
Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
27.4K
Blind Procedures
13.4K
Ideally, the people who observe and record the children’s behavior are unaware of who was assigned to the experimental or control group, in order to control for experimenter bias. Experimenter bias refers to the possibility that a researcher’s expectations might skew the results of the study. Remember, conducting an experiment requires a lot of planning, and the people involved in the research project have a vested interest in supporting their hypotheses. If the observers knew which...
13.4K
Blinding
3.8K
Blinding is a commonly used method of not telling participants which treatment a subject is receiving. Blinding is a critical part of a randomized control trial or RCT. It reduces the bias that affects the results. In an RCT, blinding is used in the form of a placebo. A placebo effect occurs when untreated subjects falsely believe they have received the treatment and report improved symptoms. A placebo or a dummy treatment is administered to subjects to negate the bias caused by such an effect.
3.8K
Group Therapy
391
Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
391


