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PANKAJ MALHOTRA, MDChandigarh, India
• Associate Professor, Department of Internal Medicine Pgimer, Chandigarh, India
• Dr. Pankaj Malhotra received his medical degree from Punjab University and his training in internal medicine at PGIMER, Chandigarh, India. He has several invited and peer reviewed publications. Dr. Malhotra was awarded the CML Fellowship by International CML Foundation (iCMLF) to Knight Cancer Centre, Oregon Science and Health University, Portland, USA. Areas of Interest include leukemia, stem cell transplant and myeloma.
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Novel treatmentsPankaj Malhotra, MD, FACP
Professor of Clinical Hematology
Dept. of Internal Medicine, PGIMER, Chandigarh, India
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Normal production of globinchains
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Hallmark of b thal
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Produce b chains:1. Stem celltransplantation2. Gene therapy3. antisense molecules to silence mRNA splicing4. Induced pluripotent stem cells
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Increase the g chain production:1. Drugs2. Gene therapy
Decrease a chain production
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Gene therapy
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1. Selection of virus2. Technique
1. How long will it work
2. Long term side
effects
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1. Selection of virus2. Technique
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1. Selection of virus2. Technique
1. How long will it work
2. Long term side
effects
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iPSC
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Caution!
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Silence Hb F suppression
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Increase the g chain production
1. Drugs2. Gene therapy
Silence Hb F suppression
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Drugs for increasing Hb F
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Other drugs for increasing Hb F
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Conclusions on novel (not so) treatments in thalassemia
b globin production
• Gene therapy
• Antisense molecules to alter RNA splicing
• iPS cells
Upregulation of gamma globin production
• Drugs (HbF production)
• Upregulation of genes
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