Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy. Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Question

Want to see the full answer?

Answer 1

Question

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Expert Solution & Answer

Want to see the full answer?

Check out a sample textbook solution

See solution

Students have asked these similar questions

Kinetics: One-Compartment First-Order Absorption 1. In vivo testing provides valuable insight into a drug’s kinetics. Assessing drug kinetics following multiple routes of administration provides greater insight than a single route of administration alone. The following data was collected in 250-g rats following bolus IV, oral (PO), and intraperitoneal (ip) administration. Using this data and set of graphs, determine:(calculate for each variable) (a) k, C0, V, and AUC* for the bolus iv data (b) k, ka, B1, and AUC* for the po data c) k, ka, B1, and AUC* for the ip data (d) relative bioavailability for po vs ip, Fpo/Fip (e)absolute ip bioavailability, Fip (f) absolute po bioavailability, Fpo

3. A promising new drug is being evaluated in human trials. Based on preliminary human tests, this drug is most effective when plasma levels exceed 30 mg/L. Measurements from preliminary tests indicate the following human pharmacokinetic parameter values: t1/2,elim = 4.6hr, t1/2,abs = 0.34hr, VD = 0.29 L/kg, Foral = 72%. Based on these parameters, estimate the following if a 49 kg woman were to receive a 1000mg oral dose of this drug: (a) Estimate the plasma concentration of the drug at 1hr, 6 hr, and 20hr after taking the drug ( Concentration estimate) (b) Estimate the time for maximum plasma concentration (tmax). (c) Estimate the maximum plasma concentration (Cmax). (d) Estimate the time at which the plasma level first rises above 30 mg/L. (Note this is a trial and error problem where you must guess a time, plug it into the concentration equation, and determine if it is close to 30 mg/L. Hint: based on part (a) it should be apparent that the answer is less than 1hr.) (e)…

List substitutions in your diet you could make to improve it based on what you know now about a balanced diet. For instance, if you like to drink soda, you might substitute skim milk or water for some of the soft drinks you consumed. List the item you wish to replace with the new item and what you hope to accomplish with that substitution. Be sure to choose foods you know that you'd enjoy and you consider more "healthful." If you feel your diet is already balanced, describe how you accomplish your balanced intake and when you began eating this way.

Answer 2

Question

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Expert Solution & Answer

Want to see the full answer?

Check out a sample textbook solution

See solution

Answer 3

Question

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Expert Solution & Answer

Want to see the full answer?

Check out a sample textbook solution

See solution

Answer 4

Question

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Expert Solution & Answer

Want to see the full answer?

Check out a sample textbook solution

See solution

Answer 5

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Answer 6

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Answer 7

Chapter ST.5, Problem 6RQ

Summary Introduction

To determine: Whether the therapeutic gene delivered to Ashanti replaced the defective copy of the ADA gene at the time of the first successful trial of gene therapy.

Introduction: The FDA approved the first human gene therapy trial in 1990. This therapy started with the treatment of a young girl named Ashanti De Silva. She has a heritable disorder called adenosine deaminase severe combined immunodeficiency (ADA-SCID). SCID is a hereditary disease that is a result of many genetic mutations, such as the abnormal development of B and T cells.

Answer 8

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Answer 9

Summary Introduction

To determine: The reason for choosing white blood cells as the targets for the therapeutic gene.

Introduction: ADA-SCID is a condition that is affecting nearly 1-9 out of every 1 million live births. Individuals suffering from SCID lack a normal immune system. They generally die from minor infections.

Answer 10

Expert Solution & Answer

Answer 11

Expert Solution & Answer

Answer 12

Want to see the full answer?

Check out a sample textbook solution

See solution

Answer 13

Ch. ST.1 - What is the difference between innate immunity and...Ch. ST.1 - What evidence demonstrates that CRISPR-Cas is an...Ch. ST.1 - Prob. 3RQ Ch. ST.1 - Why was the type II CRISPR-Cas9 system of S....Ch. ST.1 - Prob. 5RQ Ch. ST.1 - What is a single guide RNA, and what role does it...Ch. ST.1 - What is the difference between nonhomologous...Ch. ST.1 - Prob. 8RQ Ch. ST.1 - Prob. 9RQ Ch. ST.1 - Prob. 1DQ

Solution Summary: The author explains that the therapeutic gene delivered to Ashanti did not replace the defective copy of the ADA gene.

Concept explainers

Videos

Want to see the full answer?

Chapter ST Solutions