The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment. Concept Introduction : The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

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Answer 1

Question

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

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Question #3: In the KeyGene paper, the authors state that it would be useful if pollen from an apomict would transmit apomixis-inducing genes to the female in the cross (assuming the pollen is viable). Assuming there was just one gene conferring gametophytic obligate apomixis, and that the two parents are inbreds, what would be the consequences of such a cross if: a) The apomixis was a dominant trait? Indicate the genotypes and phenotypes (apomict or non- apomict) of the parents, F1 and F2 generations. Remember to include the expected genotypic and phenotypic ratios (or percentages) in the F1 and F2 generations, and to position the female first (left side) in the parental cross. b) The apomixis was a recessive trait? Indicate the genotypes and phenotypes (apomict or non- apomict) of the parents, F1 and F2 generations. Remember to include the expected genotypic and phenotypic ratios (or percentages) in the F1 and F2 generations, and to position the female first (left side) in the…

Question #5: Assume that two genes are identified that confer gametophytic facultative apomixis in soybean. The genes show independent assortment. Recessive alleles at both loci are required for the facultative apomixis. Facultative apomixis is triggered when the temperature at pollination is above 20 degrees C. At temperatures below 20 degrees C, all reproduction is sexual, independent of genotype. A facultative apomict male, capable of producing viable pollen, was crossed with a sexually reproducing female. Assuming the parents are completely inbred, what are the predicted phenotypic ratios (apomict: non-apomict) for the F1, F2, and DH (F1-derived) generations at each of the following temperatures*: a) 15°C? b) 25°C? *for full credit, show crosses and genotypes where appropriate. Remember to position the female first (left side) in the cross. Type your answer here:

Answer 2

Question

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

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Answer 3

Question

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

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Answer 4

Question

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

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Answer 5

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

Answer 6

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

Answer 7

Chapter 19, Problem 24COQ

Summary Introduction

To determine:

The characteristics of blindness that make LCA an attractive candidate for gene therapy treatment.

Concept Introduction:

The insertion of the genetic material (DNA) into the cells of patient (human) for the treatment of particular disorder is known as gene therapy. The procedure of the gene therapy includes a patient who is given healthy genes to make up for the diseased gene or faulty gene.

Answer 8

Expert Solution & Answer

Explanation of Solution

Researchers have suggested that half of blindness cases in adults before age 45 are due to heredity. Because of nerves associated with eyes, brain parts involved in visual perception and complexity of eye, defects could occur in various genes that may result in blindness. Leber congenital amaurosis (LCA) is a form of blindness which is inherited to offspring by parents as autosomal recessive disorder.

LCA occurs when there is a defect or mutation in one of the genes responsible for functioning and development of photo receptors cells in retina. Children with two defective copies of gene begin to lose eyesight at birth as light sensing cells die and become blind before the age of 40. There is another type of LCA which is caused by defective RPE65 gene. This gene is responsible for converting vitamin A into a light absorbing pigment called rhodopsin.

LCA disorder can be treated by the help of gene therapy. The characteristics of retinal blindness such as LCA that makes it suitable for gene therapy are:

(1) Retina consists of an ordered layer of epithelial cells which make it easier for administrating therapeutic gene containing DNA vector in eye cell population.

(2) The retinal tissue is smaller than visceral organ. This feature of retinal tissue permits a targeted and efficient approach for insertion of DNA vector inside cells.

(3) The intraocular environment is separated or isolated from immune system by the blood-retinal barrier. It is important to prevent any extreme immune response on insertion of foreign antigen.

(4) The very slow rate of retinal cells division makes it possible for using non-integrating vectors which facilitate sustained expression of transgene used in gene therapy.

Answer 9

Expert Solution & Answer

Answer 10

Expert Solution & Answer

Answer 11

Conclusion

The characteristics of retinal cells such as compact epithelial layer, small retinal tissue, separation of intraocular environment from immune system and slow rate of retinal cell division, makes treatment of retinal blindness such as LCA suitable for gene therapy.

Answer 12

Ch. 19.1 - Prob. 1COQ Ch. 19.2 - Prob. 10COQ Ch. 19.2 - Prob. 11COQ Ch. 19.2 - Prob. 1CC Ch. 19.2 - Prob. 25AQP Ch. 19.2 - Prob. 26AQP Ch. 19.2 - Prob. 27AQP Ch. 19.2 - Prob. 28AQP Ch. 19.2 - Prob. 29AQP Ch. 19.2 - Prob. 2CC

Explanation of Solution

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Chapter 19 Solutions