HELLO STEM CELL

ENTHUSIAST
HELLO STEM CELL ENTHUSIAST

WELCOME OUR SUPERHERO

STEM CELL AND
GENE THERAPY
SANJEEDA ISLAM
MSC BIOTECHNOLOGY PART 1
31031623007
 INTRODUCTION
• STEM CELL THERAPY AND GENE THERAPY ARE
TWO SEPARATE METHODOLOGIES

• USED TO TREAT GENETIC DISORDERS

• COMBINED TOGETHER TO TAKE FULL ADVANTAGE

OF BOTH METHODS
 STEM CELL THERAPY
STEM CELLS ARE PRIMITIVE CELLS
WITH THE CAPACITY TO SELF-
RENEW AND THE ABILITY TO
DIFFERENTIATE INTO DIFFERENT
CELL TYPES.

TYPES:

• TOTIPOTENT STEM CELLS

• PLURIPOTENT STEM CELLS

• MUTIPOTENT STEM CELLS

• UNIPOTENT CELLS

FIG NO. 1. STEM CELL DIFFERENTIATION

 GLOBAL ONGOING CLINICAL TRAILS OF STEM CELL
BASED THERAPIES FOR VARIOUYS DISEASES LIKE:

• PAPARKINSON’S DISEASE
• SPINAL CORD INJURY
• HEART FAILURE
• HAEMATOLOGICAL DISEASES
• CANCER
• ARTHRITIS
• DIABETES
• PERIPHERAL VASCULAR DISEASE

FIG NO. 3. PROCESS OF STEM CELL THERAPY

GENE THERAPY
GENE THERAPY

FIG NO. 4. GENE THERAPY

GENE THERAPY
COMBINATION STRATEGY
• Genetic modification in stem cells will be
designed either to completely correct the
genetic defect, or at least to compensate for
the genetic defect.

• Let’s discuss the combination of stem cell

and gene therapy by using example of its
application
 APPLICATIONS
KOHN DEVELOPED A SUCCESSFUL BLOOD STEM CELL GENE
THERAPY

FOR VARIOUS DISEASES LIKE :

• Sickle cell disease
• severe combined immunodeficiency due to adenosine deaminase deficiency
(ADA-SCID)
• rare immune disorders like X-linked SCID
• leukocyte adhesion deficiency-I
• X-linked chronic granulomatous disease

FIG NO. 7 . DONALD KOHN Gene therapy for paediatric HIV/AIDS using bone marrow stem cells
(Distinguished professor of microbiology,
immunology and molecular genetics, paediatrics,
and molecular and medical pharmacology at
the David Geffen School Of Medicine at UCLA)
 SICKLE CELL DISEASE
Caused by a single point mutation of nucleotide
substitution

FIG NO. 8. SICKLE CELL DISEASE

FIG NO. 9. PROCESS OF COMBINED THERAPY
 ADENOSINE DEAMINASE DEFICIENCY - SEVERE COMBINED
IMMUNODEFICIENCY (BLOOD STEM CELL GENE THERAPY)

ADA-SCID is caused Blood stem cells

by mutations in the collected from
patient and cultured
ADA gene that create in laboratory

the enzyme
adenosine Genetic modification
using modified
deaminase, which is lentivirus in vitro
essential to a
functioning immune
system Injecting back in
FIG NO. 10. DAVID VETTER
patients
(1971- 1984)
“BUBBLE BOY”
 ADENOSINE DEAMINASE DEFICIENCY - SEVERE COMBINED
IMMUNODEFICIENCY (BLOOD STEM CELL GENE THERAPY)

1.UCLA :

• Successfully treated 48
out of 50 children born
with (ADA-SCID).
• Untreated patients should
take ADA enzyme
injection once or twice a
week
 IN VITRO GENE THERAPY FOR BLOOD
DISEASES:
• In blood diseases like leukemia,
gene therapy can be applied to
specific cell types, such as T
cells, NK cells, or hematopoietic
stem cells.
• By introducing therapeutic genes
(such as chimeric antigen
receptors), researchers aim to
enhance disease-specific immune
responses

FIG NO. 11. CAR T CELL THERAPY

 ADVANTAGES
• Combination of stem cell and gene therapy is more effective than individual
therapies.
• Modified cells act as factories for transgene production.
• Allows selection of cells with transgene and sufficient therapeutic agent
production.
• Provides expression-controllable cell line.
• Stem cells like HSCS, MSCS, NSCS, myoblasts, and osteoblasts studied as
gene-delivery candidates.
 LIMITATIONS

Gene Therapy Risks and Concerns

• Potential immune and inflammatory responses from host.

• Toxicity from over or under-expression of transgene.
• Risk of insertional mutagenesis in integrating viral vectors.
• Fear of virus vector regaining disease-causing ability once inside
the patient
 FUTURE PROSPECTS AND CRITIQUE

• Stem cell and gene therapies have revolutionary potential for

treating and curing diseases.
• Treating disease by digging into the cause of disease.
• Earlier only used in hereditary and genetic disease. But now can
be applicable to non hereditary disease’s like cancer, tissue
damage. (CAR-T cells)
• Challenges or limitations should be the major concern.
 CONCLUSION

• Barriers can be solved by various collaborations and critical

thinking.
• Stem cell gene therapy brings optimism among researchers,
doctors, and patients, reviving hope in the future.
• Combination strategy shows enormous promise for
revolutionizing medicine and improving lives.
 LIST OF PICTURES
FIG NO NAME
1 STEM CELL DIFFERENTIATION
2 TYPES OF STEM CELLS
3 PROCESS OF STEM CELL THERAPY
4 GENE THERAPY
5 GENE DISORDERS
6 GENE THERAPY APPROACHES
7 DONALD KOHN
8 SICKLE CELL DISEASE
9 PROCESS OF COMBINED THERAPY
10 DAVID VETTER
11 CAR T CELL THERAPY
 REFERENCES
1. Rafi M. A. (2011). Gene and stem cell therapy: alone or in combination? BioImpacts : BI, 1(4), 213–218.
https://doi.org/10.5681/bi.2011.030
2. Nadig R. R. (2009). Stem cell therapy - Hype or hope? A review. Journal of conservative dentistry : JCD, 12(4), 131–138.
https://doi.org/10.4103/0972-0707.58329 Sayed, N., Allawadhi, P., Khurana, A., Singh, V., Navik, U., Pasumarthi, S. K., Khurana, I.,
Banothu, A. K., Weiskirchen, R., & Bharani, K. K. (2022). Gene therapy: Comprehensive overview and therapeutic applications. Life
sciences, 294, 120375. https://doi.org/10.1016/j.lfs.2022.120375
3. Li, S., Tang, H., Li, C., Ma, J., Ali, M., Dong, Q., ... & Sun, C. (2023). Synthetic biology technologies and genetically engineering
strategies for enhanced cell therapeutics. Stem Cell Reviews and Reports, 19(2), 309-321.
https://doi.org/10.1007/s12015-022-10454-5
4. Papanikolaou, E., and Bosio, A. (2021) The Promise and the Hope of Gene Therapy. Front. Genome Ed. 3:618346.
https://doi.org/10.3389/fgeed.2021.618346
5. Davis, B. R., & Prokopishyn, N. L. (2009). Stem Cell Gene Therapy. In Essentials of Stem Cell Biology (pp. 591-597). Academic Press.
https://doi.org/10.1016/B978-0-12-374729-7.00065-2
THANK
YOU