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#blood clotting

12 public questions tagged with this topic.

Gene Therapy

Hemophilia gene therapy targets deficiency of:

Hemophilia A 1 in 5000 males and B 1 in 30000 males result null mutations coagulation cofactors FVIII 280 kDa glycoprotein encoded F8 Xq28 domains A1 A2 B A3 C1 C2 and FIX vitamin K dependent serine protease encoded F9 Xq27 gamma carboxyglutamic acid residues binding calcium phospholipid. Both assemble intrinsic tenase complex FVIIIa cofactor enhancing FIXa protease activity 200000-fold toward FX activation on phosphatidylserine-rich platelet surface accelerating thrombin burst fibrin clot formation. Deficiency prolongs aPTT causing hemarthrosis, muscle hematoma. Replacement requires frequent IV infusions half-life FVIII 12h FIX 18h. Gene therapy delivers codon-optimized B-domain-deleted F8 central 908 aa dispensable removed reducing size 4.4 kb fitting AAV, or FIX Padua variant R338L leucine substitution arginine 338 increasing specific activity 8-fold improving secretion. Hepatotropic AAV5 AAV6 liver-specific promoter HLP transcribe hepatocytes synthesize factor secreting into sinusoids restoring plasma levels 5-150 percent converting severe to mild reducing annualized bleeding rate 90 percent in trials Hemgenix Roctavian leading to regulatory approvals.

Ref: NEJM Hemophilia Gene Therapy Nathwani 2022; FDA Hemgenix Roctavian Approvals; Alberts Cell Biology Coagulation Cascade Chap 20.

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Tissue

Which blood component is essential for clotting?

Answer: C) Platelets. For Tissue-2, once you lock onto the key mechanism or definition, Platelets is the clear fit. If you restate the concept in your own words, Platelets is the option that correctly names the structure, process, or principle asked for in Tissue-2. So Platelets is the clean, accurate selection. If a similar stem appears later, start from the same core fact and you will land on the same kind of answer. In class notes, highlight this same phrase next to the related diagram so the wording and the picture reinforce each other. When you practise, cover the choices first, write Platelets from memory, then reveal the letter — that habit builds real recall for Tissue-2.

Ref: Animal physiology is Unit 7 of the CSIR NET Life Science Syllabus, covering core body systems. It accounts for roughly 10% of the total marks in Sections B and C.

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