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Current Pediatric Research| Volume: 22
November 28-29, 2018 | Dubai, UAE
15th World Congress on
Pediatrics, Clinical Pediatrics and Nutrition
28th International Conference on
Nursing Practice
Joint Event
&
Hemophilia A and B are the X-linked genetic disorders,
presenting with the low levels in coagulation factor VIII
(FVIII) and factor IX (FIX) leading to recurrent bleeds andmultiple
complications 1. Hemophilia is prevalent throughout world & in
seen in all the racial groups. Thousands of children in Middle
eastern countries are suffering from these crippling disorders.
These children are forced to be away from the routine activities
of their age fellows spending most of the time in hospitals 2.
Present management protocols for hemophilia concentrate
on the intravenous infusion of clotting factor concentrates.
However, this approach is far away frompermanent cure. Newer
approach for curing the disease is the development of gene-
based therapies for hemophilia in order to attain sustained high
levels of serum clotting factors to correct the bleeding diathesis.
Different types of viral and non-viral genetic rearrangements
to a range of target cells in the body, i.e. hematopoietic stem
cells, hepatocytes 3, skeletal muscle cells, and endothelial cells,
have been considered for possible hemophilia gene therapy. So
far the most promising vectors are Adeno-associated virus and
lentiviruses. The results of gene therapy were very satisfactory
in murine and canine laboratory models and these promising
studies encouraged clinical trials in human suffering from
this debilitating disorder. Consistently high levels of clotting
factors VII and IX were observed in initial clinical trials resulting
in long term avoidance of infusions of deficient factors in the
patients. But some the corner stones are yet to be turned as
it has been observed that the gene-modified cells are a potent
target for immune exposure to effector T cells, resulting in
recurrence of bleeding episodes 4. More effectivemeasures are
needed to achieve a full hemostatic correction in our children
presenting with either hemophilia A or B. Once fully developed
and free of all the hazard, it would be like bright sun shine
in darkened lives of Hemophilia patients and their families.
Speaker Biography
Tahir Jameel is working as professor of Hematology in King Abdulaziz University,
Jeddah Saudi Arabia. He has a vast teaching experience of Clinical and academics in
Hematology. He has published many research articles in national and international
medical journals. He is coauthor of three medical books, has written many chapters
is multiauthor books. He has vast exposure in Pediatric Hematology. especially in
managing Thalassemia, Hemophilia other hemoglobinopathies.
e:
Tahir Jameel
King Abdulaziz University, Saudi Arabia
Gene Therapy-A hope for Hemophilia Children
Tahir Jameel, Pediatrics and Clinical Pediatrics 2018
& Nursing Practice 2018, Volume 22
DOI: 10.4066/0971-9032-C2-004