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関連する概念動画

Gene Therapy00:59

Gene Therapy

27.7K
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...
27.7K
Group Therapy01:26

Group Therapy

460
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...
460
Gene Flow02:39

Gene Flow

38.1K
Gene flow is the transfer of genes among populations, resulting from either the dispersal of gametes or from the migration of individuals.
38.1K
Gene Conversion02:08

Gene Conversion

10.7K
Other than maintaining genome stability via DNA repair, homologous recombination plays an important role in diversifying the genome. In fact, the recombination of sequences forms the molecular basis of genomic evolution. Random and non-random permutations of genomic sequences create a library of new amalgamated sequences. These newly formed genomes can determine the fitness and survival of cells. In bacteria, homologous and non-homologous types of recombination lead to the evolution of new...
10.7K
Gene Families01:57

Gene Families

10.0K
Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
10.0K
What is Gene Expression?01:42

What is Gene Expression?

197.1K
Overview
Gene expression is the process in which DNA directs the synthesis of functional products, that is, proteins. Cells can regulate gene expression at various stages. It allows organisms to generate different cell types and enables cells to adapt to internal and external factors.
Genetic Information Flows from DNA to RNA to Protein
A gene is a stretch of DNA that serves as the blueprint for functional RNAs and proteins. Since DNA is made up of nucleotides and proteins consist of amino...
197.1K

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関連する実験動画

Updated: Feb 12, 2026

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research
10:10

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

Published on: November 2, 2018

9.9K

網膜変性に対する遺伝子治療

Rajendra S Apte1

  • 1Washington University School of Medicine, 660 South Euclid Avenue, Box 8096, St. Louis, MO 63110, USA.

Cell
|March 24, 2018
PubMed
まとめ

アデノウイルスのベクトルを用いた遺伝子治療は正常なRPE65タンパク質を網膜細胞に提供します. この治療は,RPE65遺伝子変異によって引き起こされる遺伝性網膜変性を持つ患者の視覚サイクルと視力を回復します.

科学分野:

  • 眼科について
  • 遺伝学
  • 分子生物学

背景:

  • 遺伝性網膜変性 (IRD) は,しばしばRPE65遺伝子のバイアレル変異によって引き起こされます.
  • これらの変異は視覚のサイクルを乱し 漸進的な視力喪失につながります

研究 の 目的:

  • RPE65に関連するIRDのための新しい遺伝子治療を開発し,評価する.
  • 遺伝子の増強によって 視覚のサイクルと視覚機能を 回復させるため

主な方法:

  • 正常なヒトRPE65遺伝子を有するアデノウイルスベクターの 網膜細胞への手術.
  • 視覚サイクル回復と視覚機能の回復の評価

主要な成果:

  • 遺伝子療法は機能的なRPE65タンパク質を網膜細胞に成功させた.
  • 視力の回復と部分的な回復が観察されました.

結論:

  • アデノウイルスベクター媒介のRPE65遺伝子治療はRPE65関連IRDの治療に有効なアプローチです.
  • この"ベンチ・トゥ・ベッドサイド"療法では 患者に視力を回復させる可能性があります

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Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model
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Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model

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関連する実験動画

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Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium
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Published on: August 7, 2015

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Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model
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Transpupillary-Guided Trans-Scleral Transplantation of Subretinal Grafts in a Retinal Degeneration Mouse Model

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