International Conference On Orphan Drugs and Rare Diseases

August 17-19, 2023

August 17 -19, 2023 | Online Event

Gene and Stem Cell Therapy

Gene and Stem Cell Therapy

The insertion of genetic material into cells for therapeutic purposes is known as gene therapy. Stem cells are cells that can self-renew indefinitely and have the ability to differentiate into intermediate and mature cells. Adult stem cells, also known as tissue stem cells, create cells that are unique to the tissue in which they are located. They are relatively unspecialized, and when they differentiate, they are predestined to give rise to specific cell types. Different stem cell populations may be limited to specific developmental stages or cell types. The initial goal of gene therapy was to treat inherited genetic disorders by replacing a mutated gene with a functional one. On the other hand, stem cells were largely used for tissue repair. However, these technologies have a lot of overlap in their applications. In the treatment of hereditary and acquired disorders, both gene and stem cell therapies can be used. When two techniques are combined, their therapy potential is maximized. Gene therapy and stem cell research have grown in prominence in recent years.

  • Hematopoietic Stem Cells
  • Mesenchymal Stem Cells
  • Neural Stem Cells
  • Myoblasts
  • Osteoblasts
  • Limitations
  • Control Over Gene Expression
Orphan Drugs 2023 Speakers
Speaker at Orphan Drugs and Rare Diseases 2023 - Vicki Ratner

Vicki Ratner

ESSIC - International Society for the Study of BPS, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Kondakova O. B

Kondakova O. B

National Medical Research Center for Children's Health Federal state autonomous institution of the Russian Federation Ministry of Health, Russian Federation
Speaker at Orphan Drugs and Rare Diseases 2023 - Paul L Kaufman

Paul L Kaufman

University of Wisconsin-Madison, United States
Speaker at Orphan Drugs and Rare Diseases 2023 - Magali Taiel

Magali Taiel

GenSight Biologics, France

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