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Stem Cells & Genome Therapies (BIO330) | 80+ Verified Q&As on iPSCs, Organoids, AMD, GMP, RPE Cells

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This comprehensive document contains over 80 expert-verified exam questions and answers covering key concepts in Stem Cells and Genome Therapies, ideal for students enrolled in BIO330 or related biomedical and molecular biology programs. It provides deep insights into stem cell properties, therapeutic development, and genome manipulation technologies through structured, exam-style content. Topics include: Stem cell biology: inner cell mass, self-renewal, pluripotency, fetal stem cells, and Waddington’s epigenetic landscape iPSCs & reprogramming: detailed protocols involving Oct4, Sox2, Klf4, and c-Myc; their use in disease modeling and drug screening Retinal therapies: RPE cell biology, AMD pathogenesis, retinal organoid development, and results from hESC-derived RPE clinical trials (e.g., the London Project Trial) Hearing regeneration: cochlear hair cell repair, Rincell-1 therapy, ABR testing Organoids & disease models: CEP290 retinal organoids, Stargardt disease, and CRISPR/Cas9 editing for IRDs Gene expression control: ASOs (e.g., QR-110), AON modifications, morpholino therapies for LCA10, and gene-agnostic vs gene-specific treatments Regulatory frameworks: GMP, QMS, CMC, AD, PD, ATP, and the roles of CMOs/CDMOs in therapy development Also included are case-based examples of ocular therapies (SLET, COMET), zebrafish regeneration models, CRISPR repair mechanisms, and sequencing technologies (Sanger, NGS, WES, WGS, long-read platforms). Best suited for: Students in regenerative medicine, biomedical sciences, molecular genetics, and stem cell biology Medical and health science students preparing for advanced therapeutics and gene therapy exams Research trainees, lab techs, and clinical fellows working on therapeutic development, genome editing, or regulatory compliance Professionals aiming to understand GMP manufacturing and analytical validation in cell and gene therapy contexts Keywords: stem cells, iPSCs, Waddington landscape, organoids, RPE cells, retinal degeneration, AMD, CRISPR, ASO, morpholinos, Fuchs dystrophy, CTG18.1, GMP, QMS, CMC, cell therapy, regenerative medicine, retinal therapy, auditory regeneration, SLET, Stargardt disease, ABCA4, CAR-T therapy, iPSC reprogramming

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Institution
Cell Biology
Course
Cell Biology

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Stem Cells & Genome Therapies 2026
Expert Verified | Ace the Test



Inner cell mass - 🧠ANSWER ✔✔- One of the two first lineages originated

from the fertilised embryo.

- The ICM gives rise to all embryonic tissues and the yolk sac.


Stem cells properties - 🧠ANSWER ✔✔self renewing and potency


Fetal stem cells - 🧠ANSWER ✔✔Cord blood sc, amniotic fluid sc.


Waddington epigenetic landscape - 🧠ANSWER ✔✔Depicts how a cell

progresses from an undifferentiated state to one of a number of discrete,

distinct, differentiated cell fates during development

,James A thomson, Joseph Itskovitz-Eldor, et al. - 🧠ANSWER

✔✔Embryonic stem cell lines derived from humman blastocyst


HESC derivation methods - 🧠ANSWER ✔✔Immunosugical method


Whole/partial embryo culture method

Immunosurgical derivation method (advantages and disadvantages) -

🧠ANSWER ✔✔+


- Easy isolation of ICM from blastocysts possessing large and distinct ICM.

- Selective removal of trophectoderm from expanded blastocysts.

-

- Possibility of contamination with animal pathogens.

- Difficult to isolate ICM from blastocysts having small or indistinct ICM.

Whole embyo derivation method (advantages and disadvantages) -

🧠ANSWER ✔✔+


- Can be used regardless of blastocyst quality.

- No loss of ICM during the process.

- Alleviates contamination with pathogens.

, -

- Risk of trophectoderm overgrowth (impeded growth of ICM).

Partial embyo derivation method (advantages and disadvantages) -

🧠ANSWER ✔✔+


- Efficient for blastocyst with large or small ICM.

- Minimises trophectoderm growth.

- Alleviates contamination with pathogens.

-

- Difficult to isolate portion containing the ICM.


Distinctive pluripotency markers - 🧠ANSWER ✔✔Oct4 and Nanog


HESC culture medium: lab prepared and chemically defined (give pros and

cons for both) - 🧠ANSWER ✔✔Lab prepared:


+ Can adapt media to suit needs.

- Potential batch issues with components.

- Takes time to prepare.

- Daily feeding.


3
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Institution
Cell Biology
Course
Cell Biology

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Uploaded on
January 23, 2026
Number of pages
25
Written in
2025/2026
Type
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Questions & answers

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