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Most early gene therapy studies were focused on haemophilia B, despite the fact that haemophilia B accounts for only 20 % of all haemophilia cases.

Most early gene therapy studies were focused on haemophilia B, despite the fact that haemophilia B accounts for only 20 % of all haemophilia cases. Correct Answer True

The above statement is true. Most early gene therapy studies were focused on haemophilia B, despite the fact that haemophilia B accounts for only 20 % of all haemophilia cases. Full length factor VIII cDNA (7 kb) exceeds the packaging capacity of AAV vectors (∼4.7 kb). The full length of factor IX cDNA is ∼1.6 kb.

Related Questions

Which of the following statements about gene therapy are correct?
P. Affected individuals, but not their progeny, can be cured through germline gene therapy.
Q. Affected individuals, as well as their progeny, can be cured through germline gene therapy.
R. Affected individuals, but not their progeny, can be cured through somatic gene therapy.
S. Affected individuals, as well as their progeny, can be cured through somatic gene therapy.
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Consider the following statements Gene Therapy 1. Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect. 2. CRISPR - 9 is a kind of gene therapy tool used in gene editing technology. Select the correct answer using the code given below:
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The logic problems in this set present you with three true statements: Fact 1, Fact 2 and Fact 3. Then you are given three more statements (labelled I, II, III), and you must determine which of these, if any, is also a fact. One or two of the statements could be true; or none of the statements could be true. Fact 1: Jessica has four children. Fact 2: Two of the children have blue eyes and two of the children have brown eyes. Fact 3: Half of the children are girls. If the fist three statements are facts, which of the following statement/(s) must also be a fact? I. At least one girl has blue eyes. II. Two of the children are boys. III. The boys have brown eyes. Choose the correct answer from the options given below:
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ICMR published "National Guidelines for Gene Therapy Product Development and Clinical Trails". Which of the following statements are correct about Gene Therapy Product (GTP)? 1. It is defined as any biological entity, having the required gene, that could introduce modifications in the genome for therapeutic benefit. 2. GTPs work by repairing, replacing or deactivating dysfunctional disease-causing genes to restore normal function. 3. As per the New Drugs and Clinical Trial Rules (2019), the GTPs falls under 'new drug' and shall always be deemed as 'new drug'. 4. Thalassemia, Gaucher's disease, Sickle cell anaemia and Haemophilia fall under the genetically inherited disease category. Select the correct answer using the code given below:
A,B,C,D,E,G and I are seven friends who study in three different standards namely 5th, 6th and 7th such that not less than two friends study in the same standard. Each friend also has a different favorite subject namely History, Civics, English, Bangla, Science , Maths , and Economics but not necessarily in the same order. A likes Maths and studies in the 5th standard with only one other friend who likes Bangla. I studies with tow other friends . Both the friends who study with I like languages (Here languages include only Science , English and Bangla ) . D studies in the 6th standard with only one person and does not like Civics . E studies with only one friend . The one who likes history does not study in 5th or 6th standard . E does not like languages. C does not like English , Science or Civics. Which combination represents E's favorite subject and the standard in which he studies?