Yes, Adenosine Deaminase (ADA) deficiency can be treated by bone marrow transplantation (Option 2).
However, other possible treatments include:
Enzyme Replacement Therapy (ERT): Injecting ADA enzyme into the patient.
Gene Therapy: Introducing a functional ADA gene into the patient's cells using a viral vector.
Bone marrow transplantation is a potential permanent cure if a suitable donor is available.
Bone marrow transplantation, also known as hematopoietic stem cell transplantation, is the primary curative treatment for ADA deficiency.
The procedure involves replacing the patient's faulty bone marrow cells with healthy stem cells from a compatible donor. The transplanted stem cells, which contain the functional ADA gene, can produce normal ADA enzyme activity and restore immune function in the patient.
The reason bone marrow transplantation is effective in treating ADA deficiency is that bone marrow is the site of hematopoiesis, where blood cells, including immune cells, are produced. By replacing the defective bone marrow cells with healthy ones, the patient receives a source of functional ADA enzyme, which helps in the proper development and functioning of immune cells.
List I | List II | ||
A | Down’s syndrome | I | 11th chormosome |
B | α-Thalassemia | II | ‘X’ chromosome |
C | β-Thalassemia | III | 21st chromosome |
D | Klinefelter’s syndrome | IV | 16th chromosome |
Find the incorrect statement among the following: n(A) In sex-linked recessive traits, the gene is transmitted from an unaffected carrier female to some of the male progeny. n(B) Accumulation of phenylpyruvic acid in the brain results in mental retardation. n(C) Individuals affected by Down’s Syndrome will have congenital heart defects and are more intelligent. n(D) Turner’s Syndrome is caused due to the absence of one X chromosome.
List-I | List-II |
(A) Thalassemia | (I) 47, XXY |
(B) Klinefelter’s syndrome | (II) Sex-linked recessive disorder |
(C) Turner’s syndrome | (III) 45, XO |
(D) Colour blindness | (IV) Autosomal recessive disease |