/*! This file is auto-generated */ .wp-block-button__link{color:#fff;background-color:#32373c;border-radius:9999px;box-shadow:none;text-decoration:none;padding:calc(.667em + 2px) calc(1.333em + 2px);font-size:1.125em}.wp-block-file__button{background:#32373c;color:#fff;text-decoration:none} Problem 110 Assertion: ADA deficiency can be... [FREE SOLUTION] | 91影视

91影视

Log out

Learning Materials

Features

Discover

Chapter 12: Problem 110

Assertion: ADA deficiency can be cured by bone marrow transplantation. Reason: ADA deficiency can be treated by enzyme replacement therapy.

Short Answer

Expert verified
Yes, ADA deficiency can be cured by bone marrow transplantation as it introduces stem cells capable of producing ADA enzyme. Alternatively, enzyme replacement therapy treats ADA deficiency by providing the required enzyme externally, but it does not cure the deficiency.

Step by step solution

01

Understanding ADA Deficiency

ADA deficiency is a rare genetic disorder that damages the immune system, leaving individuals susceptible to serious infections. This disorder is caused by a lack of the enzyme adenosine deaminase (ADA).
02

Understanding Bone Marrow Transplantation as a Cure

Because bone marrow contains stem cells that develop into white blood cells, replacing the patient's bone marrow with healthy marrow can potentially cure ADA deficiency. The new marrow will produce cells that produce enough ADA enzyme to maintain immune functions.
03

Understanding Enzyme Replacement Therapy as a Treatment

Enzyme replacement therapy serves as a treatment not necessarily a cure. It represents a long-term treatment strategy where individuals are given an external supply of ADA enzyme, without correcting the fundamental issue of enzyme deficiency in their cells. However, this can alleviate symptoms and improve quality of life.

Unlock Step-by-Step Solutions & Ace Your Exams!

  • Full Textbook Solutions

    Get detailed explanations and key concepts

  • Unlimited Al creation

    Al flashcards, explanations, exams and more...

  • Ads-free access

    To over 500 millions flashcards

  • Money-back guarantee

    We refund you if you fail your exam.

Over 30 million students worldwide already upgrade their learning with 91影视!

Key Concepts

These are the key concepts you need to understand to accurately answer the question.

Bone Marrow Transplantation
Bone marrow transplantation (BMT) is an advanced medical procedure used to replace damaged or diseased bone marrow with healthy marrow. In the context of ADA deficiency, which impairs the immune system, BMT can be a potential cure. Here's how it works:
  • A donor with matching tissue type is identified, often a close family member, to minimize the risk of rejection.
  • The recipient's diseased marrow is eradicated using chemotherapy or radiation, making room for the new marrow.
  • Healthy bone marrow stem cells are then transplanted into the recipient's bloodstream.
  • These healthy stem cells migrate to the bone cavities and begin to produce new blood cells, including white blood cells that can produce the ADA enzyme.

It's important to note that BMT is a complex and potentially risky treatment that requires a long-term commitment to follow-up care. Success depends on several factors, including the match between donor and recipient and the recipient's condition before the transplant.
Enzyme Replacement Therapy
Enzyme replacement therapy (ERT) offers a lifeline to individuals with ADA deficiency by supplementing the missing enzyme. Unlike bone marrow transplantation, which aims to cure, ERT focuses on managing the disease:
  • Patients receive regular injections of the ADA enzyme, known as PEG-ADA.
  • The therapy aims to restore the enzyme's function in the body, allowing for the breakdown of toxins that would otherwise harm the immune system.
  • While ERT improves health and prolongs life, it's not a cure, as patients typically require ongoing treatment.

ERT is considered less invasive than BMT and does not carry the same risks of transplantation, like graft-versus-host disease. However, it demands adherence to a lifelong treatment regimen and may become less effective over time as the body might develop antibodies against the foreign enzyme.
Genetic Disorder
A genetic disorder is a disease caused by abnormalities in an individual's DNA. ADA deficiency is one such hereditary condition:
  • It results from mutations in the ADA gene, which encodes the adenosine deaminase enzyme.
  • The lack of ADA enzyme impairs the breakdown of toxic metabolites in the body, leading to the accumulation of substances harmful to white blood cells.
  • This genetic malfunction causes severe combined immunodeficiency (SCID), characterized by immune system deficiencies.

Understanding the genetic basis of ADA deficiency is crucial as it guides the approach to treatment, whether it be enzyme replacement therapy, gene therapy, or bone marrow transplantation. Moreover, genetic testing can identify carriers of the disease and support efforts in preventing its transmission.
Immune System Deficiencies
Immune system deficiencies, or immunodeficiencies, arise when components of the immune system are missing or dysfunctional. ADA deficiency leads to a specific form of immunodeficiency:
  • In ADA deficiency, the lack of the enzyme leads to cellular toxicity and destruction of white blood cells.
  • As these cells are responsible for fighting infections, their destruction results in severe vulnerability to infections.
  • Patients may experience recurrent, severe infections and may not respond well to vaccinations or antimicrobial treatments.

Managing these deficiencies requires enhancing immune system functionality, either by replacing the missing enzymes, as in ERT, or by re-establishing a functioning immune system with procedures like bone marrow transplant. These treatments are vital in providing patients with a defense mechanism against infections and a better quality of life.

One App. One Place for Learning.

All the tools & learning materials you need for study success - in one app.

Get started for free

Most popular questions from this chapter

Some of the steps involved in the production of humulin are given below. Choose the correct sequence. (1) Purification of humulin. (2) Extraction of recombinant gene product from \(\mathrm{E}\). coli. (3) Culturing recombinant \(\mathrm{E}\). coli in bioreactors. (4) Introduction of recombinant plasmid into \(\mathrm{E}\). coli. (5) Synthesis of gene for human insulin artificially. (6) Insertion of human insulin gene into plasmid. (a) \(2,1,4,3,5,6\) (b) \(1,3,5,6,2,4\) (c) \(5,6,4,3,2,1\) (d) \(3,5,2,1,6,4\)

The abbreviation 'B' in Bt-toxin stands for (a) Biotechnology (b) Biotoxin (c) Bacillus (d) Toxin released by bacterium

The use of bioresources by multinational companies and other organizations, without proper authorization from the countries and people concerned without compensatory payment, is called (a) Bioethics (b) Biopiracy (c) Bioterror (d) Bioweapon

Which of the following is based on antigen-antibody reaction? (a) PCR (b) ELISA (c) Serum analysis (d) Southern blotting

Which of the following crops are now genetically modified by Bt-toxin gene? (A) Cotton (B) Corn (C) Rice (D) Tomato (E) Potato (F) Soyabean (a) A, B and C only (b) D and E only (c) A and \(\mathrm{F}\) only (d) All of these
See all solutions

Recommended explanations on Biology Textbooks

Responding to Change

Read Explanation

Biological Organisms

Read Explanation

Cellular Energetics

Read Explanation

Reproduction

Read Explanation

Organ Systems

Read Explanation

Biological Structures

Read Explanation
View all explanations

What do you think about this solution?

We value your feedback to improve our textbook solutions.

Study anywhere. Anytime. Across all devices.