What is a reverse approach in the context of target validation?在目标验证的背景下，什么是反向方法？

Question

BlackTom AI · Accepted Answer

A reverse approach in target validation means working from the observed disease phenotype back toward the molecular cause: identify the phenotype first, then discover and validate the molecular target responsible.

1) Relevant knowledge points and concepts
- Phenotype-driven (phenotypic) versus target-based discovery: phenotypic approaches begin with a disease-relevant cellular, tissue or organismal phenotype; target-based approaches begin with a candidate molecular target.  
- “Reverse” here denotes phenotype→target discovery (sometimes called reverse pharmacology or reverse genetics in different contexts), as opposed to forward/target-first strategies.  
- Target validation in the reverse approach requires mapping the phenotype to molecular mechanisms (genetics, proteomics, pathway analysis) and then experimentally confirming that modulating the identified target affects the phenotype.

2) Step-by-step reasoning
- Step 1: Observe or define a disease phenotype (e.g., aberrant cell proliferation, specific behavioral deficit, biomarker change).  
- Step 2: Use techniques such as genetic mapping, transcriptomics, proteomics, CRISPR screens, or chemical proteomics to find molecular components associated with that phenotype.  
- Step 3: Prioritize candidate molecular targets linked to the phenotype.  
- Step 4: Experimentally validate by perturbing the candidate (knockdown/knockout, overexpression, small-molecule inhibitors) and demonstrating rescue or reproduction of the phenotype.

3) Calculations
- Not applicable.

4) Why the correct answer is correct
- Option d explicitly states the phenotype-first then target-discovery workflow, which matches the definition of a reverse (phenotype-driven) approach to target validation.

5) Why the other options are incorrect
- a: Describes high-throughput screening for drug candidates before choosing a target — not phenotype-first target discovery.  
- b: Describes phenotypic compound screening (finding bioactive compounds without known targets); this is related but focuses on compounds rather than the explicit phenotype→target discovery pathway; it doesn’t capture the step of discovering the molecular target responsible.  
- c: Describes the conventional target-based (forward) approach: start with a known molecular target and validate its disease role — the opposite of the reverse approach.

6) Conclusion
- Therefore the correct choice is d: identifying a disease phenotype first and then discovering the molecular target responsible for it, matching the provided answer.

What is a reverse approach in the context of target validation?在目标验证的背景下，什么是反向方法？单项选择题

类似问题

What would be the most suitable approach in target validation if you have a disease model and a diverse library of compounds?如果你拥有疾病模型和多样化的化合物库，靶点验证中最合适的方法是什么？

What is a forward approach in the context of target validation?在目标验证的背景下，什么是前瞻性方法？

Is it important to use diverse techniques to validate targets?

What encompasses a pharmacological approach to target validation?什么样的药理学方法用于靶点验证？

Is it important to use diverse techniques to validate targets?使用多样化技术验证靶点重要吗？

Where does target validation fit into the drug discovery pipeline?靶点验证在药物发现流程中处于什么位置？

What encompasses a pharmacological approach to target validation?

Is it important to use diverse techniques to validate targets?

更多留学生实用工具

智能学习助手

风格化写作助手

论文查重助手

文献引用助手

课堂转译助手

课堂笔记助手

Quiz搜索助手

学校历年真题

智能刷题助手

智能匹配练习

希望你的学习变得更简单