Sickle Cell and the Promise of Stem Cell Therapy
Sickle cell disease and the hope of stem cell therapies; ethics in the treatment sickle cell.
The past half century has been an era of rapid discoveries: from the humble beginnings of molecular biology, discovery of the structure of DNA, research on recombinant DNA, the discovery of the human embryonic stem cell (ESC), the completion of the Human Genome Projects, mammalian cloning and the discovery of ntESCs (nuclear transfer ESCs) by somatic cell nuclear transfer and the ethical sigh of relief when Yamanaka discovered a stem cell that could be produced from an adult skin fibroblast cell and reprogrammed to pluripotency with the added benefit of equivalence to the much debated human ESC, one that necessitated derivation from the inner cell mass of a five day old blastocyst. One discovery produced thousands more questions to be answered from the realms of many disciplines: science, medicine, ethics, religion, philosophy, law, public policy and international relations.
Definition: Sickle cell syndrome is a group of inherited disorders of hemoglobin structure that is are autosomal, recessive genetic diseases. The course of the disorders is variable among patients and even chronologically over each patient’s lifetime. Sickle cell patients have red blood cells that contain hemoglobin S (sickling or crescent shaped hemoglobin that creates abnormal blood flow in small blood vessels). SCD is caused by and altered gene that produces abnormal hemoglobin, the protein in normal red blood cells that carries oxygen throughout the body. When affected red cells lose oxygen, they collapse into a sickle, or C, shape and become stiff and sticky. Clumps of these cells block blood flow and can cause severe pain, organ damage from lack of oxygen and stroke. Anemia often develops in people with the disease because sickle cells die off quickly and bone marrow does not make new ones fast enough.” (“Blood Stem-Cell Transplant Regimen Reverses Sickle Cell Disease in
The past half century has been an era of rapid discoveries: from the humble beginnings of molecular biology, discovery of the structure of DNA, research on recombinant DNA, the discovery of the human embryonic stem cell (ESC), the completion of the Human Genome Projects, mammalian cloning and the discovery of ntESCs (nuclear transfer ESCs) by somatic cell nuclear transfer and the ethical sigh of relief when Yamanaka discovered a stem cell that could be produced from an adult skin fibroblast cell and reprogrammed to pluripotency with the added benefit of equivalence to the much debated human ESC, one that necessitated derivation from the inner cell mass of a five day old blastocyst. One discovery produced thousands more questions to be answered from the realms of many disciplines: science, medicine, ethics, religion, philosophy, law, public policy and international relations.
Definition: Sickle cell syndrome is a group of inherited disorders of hemoglobin structure that is are autosomal, recessive genetic diseases. The course of the disorders is variable among patients and even chronologically over each patient’s lifetime. Sickle cell patients have red blood cells that contain hemoglobin S (sickling or crescent shaped hemoglobin that creates abnormal blood flow in small blood vessels). SCD is caused by and altered gene that produces abnormal hemoglobin, the protein in normal red blood cells that carries oxygen throughout the body. When affected red cells lose oxygen, they collapse into a sickle, or C, shape and become stiff and sticky. Clumps of these cells block blood flow and can cause severe pain, organ damage from lack of oxygen and stroke. Anemia often develops in people with the disease because sickle cells die off quickly and bone marrow does not make new ones fast enough.” (“Blood Stem-Cell Transplant Regimen Reverses Sickle Cell Disease in