Regenerative

medicine

Presented By: Darshita sharma

Roll no: 17/IBT/046

Regenerative Medicine

• The engineering field of treatment called Regenerative

Medicine or Cell therapy refers to treatment that are
founded on the concept of producing new cells to replace
malfunctioning or damaged cells as a vihicle to treat
disease and injury
• This is especially true of diseases associated with aging
such as Alzheimers disease, Parkinson’s disease, type 2
diabetes , heart failure, osteoarthritis and aging of the
immune system known as immunosencence.
• Regenerative medicine also include the possibility of
growing tissue and organs in the laboratory and
implanting them when the body cannot heal itself.
 Stem cells as regenerative
medicine

• Stem cells are unspecialized cells

that can self-renew indefinitely
and that can also differentiate
into more mature cells with
specialized functions.
• Self-renewal – the ability to go
through numerous cycles of cell
division while maintaining the
undifferentiated state.
Healing potential of adult
stem cells
• Muscle loss
• Hair loss
• Hearing loss
• Vision loss
• Cardiovescular repair
• Joint and organ repair
• Metabolic disorder
 Major Source of stem cell

Hematopoietic Mesenchymal Tissue specific Embryonic

stem cells stem cell stem cell stem cell
Hematopoietic stem cells

• In an individual Formation of all blood cell form embryonic

to adult phase of life through Hematopoiesis process.
• All blood cells arise from a type of cell called the
hematopoietic stem cells
• Failure of Hematopoiesis leads to development of leukemia.
• Transplants of hematopoietic stem cells could replace
cancerous blood system in humans and hence cure
leukemia.
Mesenchymal stem cells

• Mesenchymal stem / multipotent stem cells (MSCs)

are capable of differentiating into multiple types of
connective tissues like bone, cartilage, muscle neuron.
• MSCs have potential utility for treating a variety of
diseases and disorders, including graft versus host
disease, organ transplantation, cardiovascular disease,
brain and spinal cord injury, lung, liver and kidney
diseases, and skeletal injuries.
Tissue specific stem cells

• Stem cell are throught to exist in many other

niches throught the body apart from the bone
marrow but are perhaps best studied in the
brain and the skin.
• NSCs as they become to be known, could be
isolated from these neurospheres and selectively
differentiated into the three main cells of the
brain neurons, glial cells and oligodendrocytes.
Tissue-specific stem
cells: Germ-line
Embryonic stem cells

• ESCs have the potential to grow indefinitely and

can differentiate into all cell types of the adult.
• ESCs are found in the inner cell mass of the
human blastocyst(64 cell), an early stage of the
developing embryo lasting from the 4th to 7th
day after fertilization.
• ESCs have the advantage over other cell types of
being truly pluripotent, i.e., ESCs have the
potential to develop into all three germ layers.
Stem cell therapies
Cardiac disease

• Cardiovascular disease causes 40% of all death in the

united states each year.
• Many treatments,including pharmacological lifestyle,
and surgical, are available for coronary heart disease, but
as yet there is no treatment other than transplantation
that can cause repair of damaged heart muscle.
• Alternatives to transplant are being considered, including
Xenotransplantation and artificial mechanical hearts but
new treatments that can repair or replace damaged tissue
in the heart are desperately needed
 Musculoskeletal disease
• Stem cell research into the
.
generation of bone and
cartilage is linked intrinsically
to tissue engineering.
• MSCs can differentiate into
both osteoblasts and
chondrocytes that a
combination of MSCs and a
suitable scaffold can rectify
bone injuries in animals.
Cell therapy
neurodegenerative disease
• Parkinson’s
• Huntington’s
• Alzheimer’s
• Amyotrophic lateral sclerosis (ALS)
• Spinal cord injury
• Multiple sclerosis (MS)
Cont… 1. Transplantation of neural stem cells
Donor: fetal brain

Approaches
to Stem Cell
Therapies For
Neurological
Disease
Type 1-diabetes

• Cell therapies that replace pancreatic β-cells in type I

diabetes and augment their effect in type II diabetes
would lighten the economical and social burden
considerably.
• The insulin producing cells obtained in such experiments
may be more closely related t nerve cells than to
endocrine cells.
• The most recent study at the time of writing claimed that
euglycemia was restored in 30% of streptozotocin treated
mice implanted with ESC derived β-cells.
• Pancreatic tissue produced from ESCs are better.
Stem cell therapy :
challenges
• How to control stem cells after injection.
• Marker identification for isolating pure SCs pool.
• Finding right expression conditions
• Different different conditions
• Different delivery system and monitoring
• Long term storage for future use
Case studies

Gene therapy successfully treats ‘Bubble Boy disease’ in children

MRC-funded scientists at the UCL Institute of Child Health have
developed a successful treatment for children with ‘Bubble boy
disease’, a rare immune disorder. These children are born with
defective immune systems, and it is also known as the ‘Bubble
boy disease’ because affected children are extremely vulnerable
to infectious diseases and some of them have become famous for
living in a sterile environment. In 2015, this work led to the launch
of Orchard Therapeutics, a spin-out company which aims to
further develop gene therapy to benefit patients with serious and
life-threatening diseases.
Successful restoration of ovarian function in a young woman
with cancer
Scientists at the MRC Centre for Reproductive Health in the
University of Edinburgh developed a new technique for
restoring ovarian function in 2016. This technique led to the
first UK woman giving birth following a transplant of her frozen
ovary tissue. 
Stem-cell derived liver models allow scientists to develop
new techniques for the potential treatment of liver disease
Scientists at the MRC Centre for Regenerative Medicine at the
University of Edinburgh have developed a new technique using
stem cells to generate a liver ‘model’. Using this model, the
team have focused on developing new techniques that could
reduce the need for liver transplants. In 2016, microRNAs to
treat drug-induced toxicity were developed, followed by stem
cell implants to treat liver disease in 2018.
THANK

YO
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