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STS MODULE 12 REVIEWER

Gene therapy involves modifying genetic material in cells to treat diseases like cystic fibrosis and certain cancers, using strategies such as gene addition, correction, silencing, and cell reprogramming. Cell therapy introduces healthy cells to repair tissues and includes various types of stem cells, each with unique properties and applications. Ethical concerns surround these therapies, particularly regarding stem cell sourcing and access, with differing regulations across countries.

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10 views

STS MODULE 12 REVIEWER

Gene therapy involves modifying genetic material in cells to treat diseases like cystic fibrosis and certain cancers, using strategies such as gene addition, correction, silencing, and cell reprogramming. Cell therapy introduces healthy cells to repair tissues and includes various types of stem cells, each with unique properties and applications. Ethical concerns surround these therapies, particularly regarding stem cell sourcing and access, with differing regulations across countries.

Uploaded by

chrizzy2024
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© © All Rights Reserved
We take content rights seriously. If you suspect this is your content, claim it here.
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STS MODULE 12 REVIEWER – GENE AND CELL THERAPHY

I. What Is Gene Therapy?

 Involves adding, removing, or modifying genetic material in a patient’s cells


to treat inherited or acquired diseases.
 Often uses a vector (commonly a non-virulent virus) to deliver DNA.
 The aim is to produce normal proteins in place of faulty ones to correct
disorders.

Diseases Gene Therapy Aims to Treat:

 Cystic fibrosis
 Hemophilia
 Congenital blindness
 ADA-SCID (Adenosine Deaminase Deficiency)
 Sickle cell anemia
 Parkinson’s, other neurological diseases
 Certain cancers
 Type 1 diabetes

Main Gene Therapy Strategies

1. Gene Addition
 Adds a working copy of a gene to compensate for a missing or faulty one.
 Used for monogenic disorders like ADA-SCID or hemophilia.
 Delivered using vectors like AAV (Adeno-Associated Virus).

Examples: ADA-SCID, Hemophilia, Leber’s Congenital Amaurosis,


Lysosomal Storage Diseases.

2. Gene Correction (Editing)


 Repairs a gene using technologies like:
 CRISPR-Cas9
 TALENs
 Zinc Finger Nucleases (ZFNs)
 Goal: Fix mutations to restore normal protein function.
 Restores proper gene function without replacing the whole gene.
 Experimental uses: HIV removal from mice genomes, Huntington’s
disease gene excision.
3. Gene Silencing
 Stops production of harmful proteins by degrading mRNA.
 Turns off genes producing harmful or excessive proteins
 Works via small RNA molecules that mimic double-stranded RNA,
triggering degradation.

Example: TNF-alpha silencing in rheumatoid arthritis.

4. Cell Reprogramming
 Converts one cell type into another by inserting/adding new genes.

Example: Turning pancreatic digestive cells into insulin-producing cells for


Type I diabetes.

5. Cell Elimination (Suicide Genes)


 Destroys cancer cells or overgrown benign cells.
 Uses viruses to deliver:
 Suicide genes (release a toxic product).
 Genes that activate the immune system or inhibit tumor blood supply.
 Oncotropic viruses: specifically target tumors.
II. Types of Gene Therapy (by target)
Type Description

Somatic Therapy Targets body cells; not inheritable

Germline Therapy Alters sperm or egg; inheritable, currently banned in humans

III. What Is Cell Therapy?

 Involves introducing healthy cells into the body to repair or replace damaged
tissues or enhance immune function.
 May be autologous (self) or allogeneic (donor).
 Includes gene-modified cells, such as CAR-T cells (engineered to kill cancer
cells).

IV. Stem Cells in Cell Therapy

Key Properties:

 Self-renewal: Can replicate without aging.


 Differentiation: Can turn into specialized cells.

Types of Stem Cells (by potency):


Type Description

Totipotent Can become all cell types + placenta (zygote stage)

Pluripotent Can become any cell except placenta (ESCs, iPSCs)

Multipotent Limited to certain lineages (e.g., blood cells from HSCs)


V. Types of Cells Used in Cell Therapy

Type of Cell Description Notes

Embryonic Stem Cells Pluripotent cells from Can become any cell type, but
(ESCs) unused IVF embryos ethical issues and rejection risk

Pluripotent, no embryo used,


Induced Pluripotent Reprogrammed adult
patient-matched but older,
Stem Cells (iPSCs) cells (e.g., skin)
mutation-prone

Patient-matched DNA + donor

Nuclear Transfer Nucleus of adult cell + mitochondria. Useful for

ESCs (ntESCs) donor oocyte mitochondrial diseases

Cloning (e.g. Dolly the sheep)

Hematopoietic Stem Found in bone marrow, blood,


Form all blood cells
Cells (HSCs) umbilical cord

Mesenchymal Stem Found in bone marrow, fat tissue,


Make bone, fat, cartilage
Cells (MSCs) umbilical cord

Neural Stem Cells Replenish brain cells Usually made from ESCs or iPSCs,
(NSCs) (neurons and glial cells) not directly extracted

Form body linings (skin, Used in corneal repair, skin and


Epithelial Stem Cells
gut) GI lining

Modified T cells (e.g., Immune cells altered for stronger


Immune Cell Therapy
CAR-T) disease-fighting power
VI. Examples of Cell Therapy Applications

 CAR-T Cell Therapy: T cells are genetically modified to target specific cancer
markers.
 HSC Transplant: Restores blood formation in leukemia.
 Epithelial SCs: Used to regenerate the cornea.
 iPSCs: Used experimentally for Parkinson’s and heart repair.

VII. Stem Cell Therapy in the Philippines

Notable Cases:

 Public figures like Gloria Macapagal-Arroyo and Juan Ponce Enrile reportedly
underwent stem cell therapy.

Government Regulation:

 Administrative Order No. 2013-0012 (DOH):


o Allows stem cell therapy only for investigative/compassionate use.
o Still undergoing clinical evaluation for:
 Blood disorders
 Cancer
 Diabetes
 Neurodegenerative diseases

Restrictions:

 Prohibited sources:
o Embryonic stem cells
o Aborted fetal cells
o Genetically modified, animal, or plant cells
 Approved sources:
o Adult stem cells
o Umbilical cord stem cells
VIII. Ethical and Global Context

Ethical Issues:

 ESCs raise debate over embryo use (some countries ban; others allow with
limits).
 Concerns over:
o Sanctity of life
o Unregulated clinics
o Equitable access

Country Positions:

| Allowed | Australia, UK, Japan, South Korea, Singapore |


| Banned | Germany, Italy, Poland, Ireland, Norway |

Common Concerns:

 Will gene therapy affect future generations?


 No, unless germline cells are altered (currently banned).
 Is gene therapy safe?
 Still under clinical trials; risks include immune reactions and off-target
effects.
 What are the ethical issues?
 Stem cell sourcing (embryos vs. adult cells).
 Access and affordability (who gets it?).
 Misleading claims by unregulated clinics.

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