My gene therapy


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  • Adult stem cells are found all over your body. Here are a few examples of places in the body with stem cells. Who here has been told that brain cells never regenerate? (hands) Whoever told you that was misinformed! Relatively recently scientists discovered that in two specific parts of your brain, neural stem cells divide and differentiate to become neurons and glial cells, which support the growth of neurons. Without neural stem cells in the hippocampus, you would probably not be able to learn or remember.The top right picture is a cross-section of the rat hippocampus, and neural stem cells are the blue dots, which divide and differentiate to form mature neurons (green) and astrocytes (red). The bottom right picture is of cultured neural stem cells (just plain blue dots), and derived from those stem cells, neurons (blue dots surrounded by red) and oligodendrocytes (blue dots surrounded by green).
  • My gene therapy

    1. 1. GENE THERAPY & STEM CELL THERAPY Dr. Gangadhar Chatterjee JR II Dept. of Biochemistry Grant Medical College, Mumbai
    2. 2. What Genes can do  Genes, which are carried on chromosomes, are the basic physical and functional units of heredity. Genes are specific sequences of bases that encode instructions on how to make proteins. it‟s the proteins that perform most life functions and even make up the majority of cellular structures.
    3. 3. Why Genetic Disorders  When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result.
    4. 4. All of us carry some defective Genes, some are apparent and many in apparent  Each of us carries about half a dozen defective genes. We remain blissfully unaware of this fact unless we, or one of our close relatives, are amongst the many millions who suffer from a genetic disease. About one in ten people has, or will develop at some later stage, an inherited genetic disorder, and approximately 2,800 specific conditions are known to be caused by defects (mutations) in just one of the patient's genes.
    5. 5. We Inherit from Parents  Most of us do not suffer any harmful effects from our defective genes because we carry two copies of nearly all genes, one derived from our mother and the other from our father. The only exceptions to this rule are the genes found on the male sex chromosomes. Males have one X and one Y chromosome, the former from the mother and the latter from the father, so each cell has only one copy of the genes on these chromosomes
    6. 6. Law of Inheritance  In the majority of cases, one normal gene is sufficient to avoid all the symptoms of disease. If the potentially harmful gene is recessive, then its normal counterpart will carry out all the tasks assigned to both. Only if we inherit from our parents two copies of the same recessive gene will a disease develop.
    7. 7. What is Gene Therapy  Gene therapy is the insertion of genes into an individual's cells and tissues to treat a disease, such as a hereditary disease in which a deleterious mutant allele is replaced with a functional one. Although the technology is still in its infancy, it has been used with some success.
    8. 8. How It Works     A vector delivers the therapeutic gene into a patient‟s target cell The target cells become infected with the viral vector The vector‟s genetic material is inserted into the target cell Functional proteins are created from the therapeutic gene causing the cell to return to a normal state
    9. 9. Gene Therapy is Experimental  Advances in understanding and manipulating genes have set the stage for scientists to alter a person's genetic material to fight or prevent disease. Gene therapy is an experimental treatment that involves introducing genetic material (DNA or RNA) into a person's cells to fight disease.
    10. 10. Majority are Trials  Gene therapy is being studied in clinical trials (research studies with people) for many different types of cancer and for other diseases. It is not currently available outside a clinical trials
    11. 11. Vivo to Vitro
    12. 12. What Gene therapy can Achieve    Replacing a mutated gene that causes disease with a healthy copy of the gene. Inactivating, or “knocking out,” a mutated gene that is functioning improperly. Introducing a new gene into the body to help fight a disease.
    13. 13. Uses of gene therapy       Replace missing or defective genes; Deliver genes that speed the destruction of cancer cells; Supply genes that cause cancer cells to revert back to normal cells; Deliver bacterial or viral genes as a form of vaccination; Provide genes that promote or impede the growth of new tissue; and; Deliver genes that stimulate the healing of damaged tissue.
    14. 14. Genes are Medicine ?  Gene therapy is „the use of genes as medicine‟. It involves the transfer of a therapeutic or working gene copy into specific cells of an individual in order to repair a faulty gene copy. Thus it maybe used to replace a faulty gene, or to introduce a new gene whose function is to cure or to favourably modify the clinical course of a condition.
    15. 15. Goal of Gene therapy     A normal gene may be inserted into a non-specific location within the genome to replace a non-functional gene. This approach is most common. An abnormal gene could be swapped for a normal gene through homologous recombination. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.
    16. 16. Delivering desired Genes
    17. 17. Gene Therapy Corrects  Gene therapy is a technique for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes:
    18. 18. Steps in Gene Therapy
    19. 19. Manipulation corrects the Defective Genes
    20. 20. Gene Therapy delivers Proteins  Today, gene therapy is the ultimate method of protein delivery, in which the delivered gene enters the body's cells and turns them into small "factories" that produce a therapeutic protein for a specific disease over a prolonged period.
    21. 21. Antisense therapy  Antisense therapy is a form of treatment for genetic disorders or infections. When the genetic sequence of a particular gene is known to be causative of a particular disease, it is possible to synthesize a strand of nucleic acid (DNA, RNA or a chemical analogue) that will bind to the messenger RNA (mRNA) produced by that gene and inactivate it, effectively turning that gene "off".
    22. 22. Antisense Therapy  Antisense therapy is not strictly a form of gene therapy, but is a genetically-mediated therapy and is often considered together with other methods
    23. 23. Newer techniqueGene silencing and RNA interference  It is a recently discovered process in cells that stops the action of specific genes by destroying mRNA and thus preventing translation of the gene product.  The diagram below summarizes the process of RNAi:  Dicer (enzyme) chops double-stranded RNA (dsRNA) into small pieces called short interfering RNA (siRNA)  siRNA combines with protein subunits to form an RNA-induced silencing complex (RISC)
    24. 24.  The siRNA within RISC unzips, exposing anticodons and thus "activating" the RISC  Activated RISC binds to target mRNA (with complementary codons)  RISC causes target mRNA to break apart, preventing translation of the gene product
    25. 25. First Approved Gene Therapy  On September 14, 1990 at the U.S. National Institutes of Health, W. French Anderson M.D. and his colleagues R. Michael Blaese, M.D., C. Bouzaid, M.D., and Kenneth Culver, M.D., performed the first approved gene therapy procedure on four-year old Ashanthi DeSilva. Born with a rare genetic disease called severe combined immunodeficiency (SCID),
    26. 26. What did they do  In Ashanthi's gene therapy procedure, doctors removed white blood cells from the child's body, let the cells grow in the laboratory, inserted the missing gene into the cells, and then infused the genetically modified blood cells back into the patient's bloodstream.
    27. 27. A success story  As of early 2007, she was still in good health, and she was attending college. Some would state that the study is of great importance despite its indefinite results, if only because it demonstrated that gene therapy could be practically attempted without adverse consequences.
    28. 28. Principles of Gene therapy     A normal gene may be inserted into a non-specific location within the genome to replace a non-functional gene. This approach is most common. An abnormal gene could be swapped for a normal gene through homologous recombination. The abnormal gene could be repaired through selective reverse mutation, which returns the gene to its normal function. The regulation (the degree to which a gene is turned on or off) of a particular gene could be altered.
    29. 29. Gene Therapy Depends on Delivery of Corrective Genes  Viral vectors are a tool commonly used by molecular biologists to deliver genetic material into cells. This process can be performed inside a living organism (in vivo) or in cell culture (in vitro). Viruses have evolved specialized molecular mechanisms to efficiently transport their genomes inside the cells they infect.
    30. 30. Viruses are used as Delivery Tolls      Viruses are used as vectors to introduce the genetic material inside the bodies. These viruses are inactivated, they are not able to reproduce Adenoviruses Herpes viruses DNA tumor viruses Retroviruses RNA tumor viruses
    31. 31. Making the new Genetic Material Functional  Gene that is inserted directly into a cell usually does not function. Instead, a carrier called a vector is used to introduce the therapeutic gene into the patient's target cells. The most common vector that is used is a virus that has been genetically altered to carry normal human DNA. Viruses cause diseases in humans by encapsulating and delivering the genes into cells.
    32. 32. Somatic and Germ Line Gene Therapy  Gene therapy can target somatic (body) or germ (egg and sperm) cells. In somatic gene therapy the recipient's genome is changed, but the change is not passed on to the next generation; whereas with germ line gene therapy the newly introduced gene is passed on to the offspring.
    33. 33. Safety  Safety: Although viral vectors are occasionally created from pathogenic viruses, they are modified in such a way as to minimize the risk of handling them.
    34. 34. Making safe Protocols   Low toxicity: The viral vector should have a minimal effect on the physiology of the cell it infects. Stability: Some viruses are genetically unstable and can rapidly rearrange their genomes. This is detrimental to predictability and reproducibility of the work conducted using a viral vector and is avoided in their design.
    35. 35. Cell type specificity   Cell type specificity: Most viral vectors are engineered to infect as wide a range of cell types as possible. However, sometimes the opposite is preferred. The viral receptor can be modified to target the virus to a specific kind of cell.
    36. 36. Lentivirus  Lentivirus (lenti-, Latin for "slow") is a genus of slow viruses of the Retroviridae family, characterized by a long incubation period. Lentiviruses can deliver a significant amount of genetic information into the DNA of the host cell, so they are one of the most efficient methods of a gene delivery vector. HIV, SIV, and FIV are all examples of lentiviruses.
    37. 37. Retroviruses  Retroviruses can infect only dividing cells. The viral genome in the form of RNA is reversetranscribed when the virus enters the cell to produce DNA, which is then inserted into the genome at a random position by the viral integrase enzyme
    38. 38. Vectors deliver the Genetic Materials  The vector, now called a provirus, remains in the genome and is passed on to the progeny of the cell when it divides.
    39. 39. Adenoviruses  As opposed to lenti viruses, adenoviral DNA does not integrate into the genome and is not replicated during cell division. Adenoviral vectors are occasionally used in in vitro experiments.
    40. 40. Choosing non infective Adenovirus  Their primary applications are in gene therapy and vaccination. Since humans commonly come in contact with adenoviruses, which cause respiratory, gastrointestinal and eye infections, they trigger a rapid immune response with potentially dangerous consequences To overcome this problem scientists are currently investigating adenoviruses to which humans do not have immunity.
    41. 41. Adeno-associated viruses  Adeno-associated virus (AAV) is a small virus which infects humans and some other primate species. AAV is not currently known to cause disease and consequently the virus causes a very mild immune response. AAV can infect both dividing and non-dividing cells and may incorporate its genome into that of the host cell. These features make AAV a very attractive candidate for creating viral vectors for gene therapy
    42. 42. Treatment using AAV  hemophilia treatments, for example, a genecarrying vector could be injected into a muscle, prompting the muscle cells to produce Factor IX and thus prevent bleeding.  Study by Wilson and Kathy High (University of Pennsylvania), patients have not needed Factor IX injections for more than a year
    43. 43. Limitation of Direct Gene Induction  The simplest method is the direct introduction of therapeutic DNA into target cells. This approach is limited in its application because it can be used only with certain tissues and requires large amounts of DNA.
    44. 44. PHYSICAL METHOD Physical approaches, including  Needle injection  Electroporation  Gene gun  Ultrasound  Hydrodynamic delivery employ a physical force that permeates the cell membrane and facilitates intracellular gene transfer 
    45. 45. Nonviral approach  Nonviral approach involves the creation of an artificial lipid sphere with an aqueous core. This liposome, which carries the therapeutic DNA, is capable of passing the DNA through the target cell's membrane
    46. 46. Nonviral Vectors: Liposome's less Immunogenic  DNA/lipid complexes are easy to prepare and there is no limit to the size of genes that can be delivered. Because carrier systems lack proteins, they may evoke much less immunogenic responses. More importantly, the cationic lipid systems have much less risk of generating the infectious form or inducing tumorigenic mutations because genes delivered have low integration frequency and cannot replicate or recombine.
    47. 47. Nanoengineered substances  Nonviral substances such as Ormosil have been used as DNA vectors and can deliver DNA loads to specifically targeted cells in living animals. (Ormosil stands for organically modified silica or silicate)
    48. 48. Transfection and Nanoengineering  Transfection is the process of introducing nucleic acids into cells by non-viral methods. The term "transformation" is preferred to describe non-viral DNA transfer in bacteria and non-animal eukaryotic cells; "transduction" is often used to describe virusmediated DNA transfer.
    49. 49. Gene Therapy should not Interfere Germ Line  The germ line of a mature or developing individual is the line (sequence) of germ cells that have genetic material that may be passed to a child  Germ line cells are immortal, in the sense that they can reproduce indefinitely. This is largely due to the activity of the enzyme known as telomerase. This enzyme extends the telomeres of the chromosome, preventing chromosome fusions and other negative effects of shortened telomeres.
    50. 50. Creating New Chromosome    Researchers are also experimenting with introducing a 47th artificial chromosome to the body. It would exist autonomously along side of the other 46, not affecting their workings or causing any mutations. It would be a large vector capable of carrying substantial amounts of genetic information and the body‟s immune system would not attack it.
    51. 51. ADA deficiency was selected for the first approved human gene therapy trial for several reasons    The disease is caused by a defect in a single gene, which increases the likelihood that gene therapy will succeed. The gene is regulated in a simple, “always-on” fashion, unlike many genes whose regulation is complex. The amount of ADA present does not need to be precisely regulated. Even small amounts of the enzyme are known to be beneficial, while larger amounts are also tolerated well
    52. 52. Problems of Large Gene  It would be a large vector capable of carrying substantial amounts of genetic code, and scientists anticipate that, because of its construction and autonomy, the body's immune systems would not attack it. A problem with this potential method is the difficulty in delivering such a large molecule to the nucleus of a target cells.
    53. 53. Gene Therapy Uses AIDS Virus to Fight AIDS  In the study, immune cells were removed from the patients' bodies, modified with a disabled AIDS virus known as a lentivirus, and then intravenously returned. The genetically altered cells disseminated antiHIV material and prevented HIV from reproducing( 07 November, 2006)
    54. 54. Cystic Fibrosis needs Correction- Gene therapy can be best option
    55. 55. Technical Difficulties in Gene Therapy  Gene delivery: Successful gene delivery is not easy or predictable, even in single-gene disorders. For example, although the genetic basis of cystic fibrosis is well known, the presence of mucus in the lungs makes it physically difficult to deliver genes to the target lung cells. Delivery of genes for cancer therapy may also be complicated by the disease being present at several sites. Gene-therapy trials for X-linked severe combined immunodeficiency (X-SCID), however, have been more successful
    56. 56. Problems with Gene Therapy  Short Lived    Immune Response     patient could have toxic, immune, inflammatory response also may cause disease once inside Multigene Disorders   new things introduced leads to immune response increased response when a repeat offender enters Viral Vectors   Hard to rapidly integrate therapeutic DNA into genome and rapidly dividing nature of cells prevent gene therapy from long time Would have to have multiple rounds of therapy Heart disease, high blood pressure, Alzheimer‟s, arthritis and diabetes are hard to treat because you need to introduce more than one gene May induce a tumor if integrated in a tumor suppressor gene because insertional mutagenesis
    57. 57. Law interferes in Gene Therapy
    58. 58. Aldo Leopold The First Bioethicist  A thing is right when it tends to preserve the integrity, stability, and beauty of the biotic community. It is wrong when it tends otherwise." Aldo Leopold, 1949, A Sand County Almanac
    59. 59. What are the ethical issues surrounding gene therapy?      How can “good” and “bad” uses of gene therapy be distinguished? Who decides which traits are normal and which constitute a disability or disorder? Will the high costs of gene therapy make it available only to the wealthy? Could the widespread use of gene therapy make society less accepting of people who are different? Should people be allowed to use gene therapy to enhance basic human traits such as height, intelligence, or athletic ability?
    60. 60. The Future of Gene Therapy  Current uses of gene therapy focus on treating or curing existing conditions. In the future, the focus could shift to prevention. As more of the human genome is understood, medicine will know more about which genes contribute to or cause disease. With that knowledge in hand, gene therapy could be used to head off problems before they occur.
    61. 61. Several Diseases have Genetic basis  Gene mutations probably play a role in many of today's most common diseases, such as heart disease, diabetes, immune system disorders, and birth defects. These diseases are believed to result from complex interactions between genes and environmental factors. When genes for diseases have been identified, scientists can study how specific environmental factors, such as food, drugs, or pollutants interact with those genes.
    62. 62. Last two decades made rapid progress  Over the last 20 years, the initial thoughts of gene therapy have been transformed into reality with more than 175 clinical trials and 2,000 patients already treated . Yet with all the trials, there is still no conclusive evidence for efficacy.
    63. 63. Attempting on Most Disabling Diseases
    64. 64. Lesch-Nyhan syndrome  The most likely candidates for future gene therapy trials will be rare diseases such as Lesch-Nyhan syndrome, a distressing disease in which the patients are unable to manufacture a particular enzyme. This leads to a bizarre impulse for selfmutilation, including very severe biting of the lips and fingers. The normal version of the defective gene in this disease has now been cloned.
    65. 65. X-linked recessive Disease  LNS is transmitted as and X-linked recessive trait. Female carriers do not show the symptoms. LNS is characterized by self-mutilating behaviours such as lip and finger biting and/or head banging. The deficiency of HPRT activity leads to accumulation of phosphoribosylpyrophos phate.
    66. 66. Lesch-Nyhan syndrome (an X-linked recessive disease)
    67. 67. Successful One Year Gene Therapy Trial For Parkinson's Disease A successful Documentation  Neurologix a biotech company announced that they have successfully completed its landmark Phase I trial of gene therapy for Parkinson's Disease.  This was a 12 patient study with four patients in each of three dose escalating cohorts. All procedures were performed under local anesthesia and all 12 patients were discharged from the hospital within 48 hours of the procedure, and followed for 12 months. Primary outcomes of the study design, safety and tolerability, were successfully met. There were no adverse events reported relating to the treatment.
    68. 68. Risks associated with current gene therapy  Viruses can infect more than one type of cells. Viral vectors may alter more than the intended cells. Or the new gene might be inserted into the wrong location in the DNA, causing cancer or other damage.    When DNA is injected directly into a tumor there is a chance that some DNA could be introduced into germ cells, producing inheritable changes. The gene might be over-expressed (toxicity); the viral vector could cause inflammation or immune reaction; the virus could be transmitted to other individuals or the environment.
    69. 69. Gene therapy of pain: emerging strategies and future directions  Gene therapy to alleviate pain could appear surprising and perhaps not appropriate when opioids and other active molecules are available. However, the possibility of introducing a therapeutic protein into some targeted structures, where it would be continuously synthesised and exert its biological effect in the near vicinity of, or inside the cells, might avoid some drawbacks of "classical" drugs.
    70. 70. Pain – Cancer a major research area  Numerous other molecules involved in pain processing or associated with chronic pain have been identified and the gene-based techniques might be particularly adapted for the evaluation of the possible therapeutic interest of these new potential targets
    71. 71. Understanding Genome and Human Genome Project is a boost to Gene Therapy
    72. 72. Do not forget Genes can be Unpredictable ?
    73. 73. Stem Cell therapy Hope stems forever
    74. 74. Importance of Stem Cell Research
    75. 75. Stem Cell History 1998 - Researchers first extract stem cells from human embryos 1999 - First Successful human transplant of insulin-making cells from cadavers 2001 - President Bush restricts federal funding for embryonic stem-cell research 2002 - Juvenile Diabetes Research Foundation International creates $20 million fund-raising effort to support stemcell research 2002 - California ok stem cell research 2004 - Harvard researchers grow stem cells from embryos using private funding 2004 - Ballot measure for $3 Billion bond for stem cells
    76. 76. Stem Cell – Definition A cell that has the ability to continuously divide and differentiate (develop) into various other kind(s) of cells/tissues
    77. 77. Stem Cell Characteristics  „Blank cells‟ (unspecialized)  Capable of dividing and renewing themselves for long periods of time (proliferation and renewal)  Have the potential to give rise to specialized cell types (differentiation)
    78. 78. Kinds of Stem Cells Stem cell type Totipotent Pluripotent Multipotent Description Examples Cells from early Each cell can develop (1-3 days) into a new individual embryos Some cells of Cells can form any (over blastocyst (5 to 14 200) cell types days) Cells differentiated, but Fetal tissue, cord can form a number of blood, and adult other tissues stem cells
    79. 79. This cell Can form the Embryo and placenta This cell Can just form the embryo Fully mature
    80. 80. Kinds of Stem Cells Embryonic stem cells • five to six-day-old embryo Embryonic germ cells • derived from the part of a human embryo or fetus that will ultimately produce eggs or sperm (gametes). Adult stem cells • undifferentiated cells found among specialized or differentiated cells in a tissue or organ after birth • appear to have a more restricted ability to produce different cell types and to self-renew.
    81. 81. Pluripotent Stem Cells – more potential to become any type of cell
    82. 82. Multipotent stem cells  Multipotent stem cells – limited in what the cells can become
    83. 83. Embryonic Stem Cells Mainly from IVF
    84. 84. Stages of Embryogenesis cleavage blastocyst 8-cell stage Blastocyst inner mass cells
    85. 85. Blastocyst Diagram
    86. 86. Adult Stem Cells An undifferentiated cells found among specialized or differentiated cells in a tissue or organ after birth • • • • • Skin Fat Cells Bone marrow Brain Many other organs & tissues
    87. 87. Induced Pluripotent Stem Cells
    88. 88. Bone Marrow  Found in spongy bone where blood cells form  Used to replace damaged or destroyed bone marrow with healthy bone marrow stem cells.  treat patients diagnosed with leukemia, aplastic anemia, and lymphomas  Need a greater histological immunocompatibility
    89. 89. Blood Cell Formation
    90. 90. Umbilical cord stem cells  Also Known as Wharton‟s Jelly  Adult stem cells of infant origin  Less invasive than bone marrow  Greater compatibility  Less expensive
    91. 91. Umbilical cord stem cells Three important functions: 1. Plasticity: Potential to change into other cell types like nerve cells 1. Homing: To travel to the site of 2. tissue damage 3. Engraftment: To unite with other tissues
    92. 92. Stem Cell Applications • Tissue repair - nerve, heart, muscle, organ, skin • Cancers • Autoimmune diseases - diabetes, rheumatoid arthritis, MS
    93. 93. Routes of stem cell delivery  INTRAVENOUS (IV) INJECTION:  very simple process and familiar to most patients.  The stem cell suspension will be administered through the IV.  Typically, no sedation is required for this procedure.  The entire IV injection process takes less than 45 minutes to complete.  The risk of infection is minimal.
    94. 94.  INTRATHECAL (IT) INJECTION:  spinal tap or lumbar puncture.  procedure used to access the cerebrospinal fluid (CSF) of the brain and spinal cord, and helps to deliver stem cells directly into the CSF, by-passing the blood-brain barrier.  least invasive method for delivering stem cells directly into the central nervous system.  CT-GUIDED INTRASPINAL CORD INJECTION: For patients with spinal cord injuries (SCI), especially diagnosed with complete SCI or incomplete SCI with severe disorder, the CT-guided intra-spinal cord injection of stem cells performed after the spine-MRI has been conducted to identify the exact injury sites.
    95. 95.  INTRAMUSCULAR INJECTION:  targeted directly into the muscles of the affected area(s).  has also been applied to treat muscular dystrophy and patients with lower limb ischemia & diabetic foot.  INTRAVASCULAR INTERVENTIONAL INJECTION:  applied to place stem cells directly into target organs or tissues using image guidance  patient is under local anesthesia.  The procedure can be applied for myocardial infarction, cardiomyopathy, liver cirrhosis and chronic renal failure, among others.
    96. 96.  INTRA-ARTICULAR INJECTION:  Patients with arthritis considered to receive stem cells through intra-articular injection.  procedure can be performed as a simple injection using a syringe directly into the cavity of the affected joints.  RETROBULBAR INJECTION:  This injection is given into the soft tissue present behind the eyeball for certain diseases like optic nerve hypoplasia, optic atrophy etc
    97. 97. Tissue Repair • Regenerate spinal cord, heart tissue or any other major tissue in the body.
    98. 98. Spinal Cord Injury Pathology at the Lesion
    99. 99. Heart Disease • Adult bone marrow stem cells injected into the hearts are believed to improve cardiac function in victims of heart failure or heart attack
    100. 100. Leukemia and Cancer • Studies show leukemia patients treated with stem cells emerge free of disease. • Injections of stem cells have also reduces pancreatic cancers in some patients. Proliferation of white cells
    101. 101. Bone Marrow Transplants to Cure Lymphomas/Thymomas  Whole Body Irradiation to remove endogenous immune system and tumor  Also total lymphoid irradiation with antithymocyte serum  Injection of bone marrow from a well matched donor to re    establish immune system Regulation of immune response to prevent graft versus host reaction. Autologous donation possible if one can purify and remove tumor cells, enriching for stem cells.. Allogeneic donors have advantage of graft versus tumor reaction to kill any remaining tumor cells. Allogeneic donors have the disadvantage of graft versus host reaction if they are not well matched.
    102. 102. Autologous vs. Allogeneic Transplants Autologous Allogenic Health y Donor Purify from Tumor cells Unfractionated bone marrow or mobilized blood Transplant Transplant Donor blood formation, graft-vstumor effect Judith Shizuru
    103. 103. Complications of Allogeneic Transplants Transplant related mortality = 10 - 15%  Regimen related toxicity  Infectious complications( bacterial,  Candida, aspergillus, HSV, CMV, Pneumocystis carini & toxoplasma)  Engraftment failure (resistance)  Graft-versus-host disease
    104. 104. Rheumatoid Arthritis • Adult Stem Cells may be helpful in jumpstarting repair of eroded cartilage.
    105. 105. Type I Diabetes • Pancreatic cells do not produce insulin • Embryonic Stems Cells might be trained to become pancreatic islets cells needed to secrete insulin.
    106. 106. Stem cells in the adult brain:
    107. 107. ICMR approved SCT in India
    108. 108. new research – reprogramming cells
    109. 109. Technical Challenges Source - Cell lines may have mutations. Delivery to target areas Prevention of rejection Suppressing tumors
    110. 110. Problems with Adult Stem Cells Mutations can lead to leukemia
    111. 111. Why is Stem Cell Research So Important to All of Us?  Stem cells can replace diseased or damaged cells  Stem cells allow us to study development and genetics  Stem cells can be used to test different substances (drugs and chemicals)
    112. 112. Why the Controversy Over Stem cells?      Embryonic Stem cells are derived from extra blastocysts that would otherwise be discarded following IVF. Extracting stem cells destroys the developing blastocyst (embryo). -Questions for ConsiderationIs an embryo a person? Is it morally acceptable to use embryos for research? When do we become “human beings?”
    113. 113. Greatly indebted to web information of PLOS online medical journal UNISTEM-INDIA STEMCELL INSTITUTESANFRANSISCO
    114. 114. Thank u all….