Scribd Logo
Upload

Welcome to Scribd!

  • 15 views

    Question: Which of The Forms of Genetic Medicine Do You Think Is The M

    Uploaded by

    Mustafa Khudhair
    Gene therapy using viral vectors for gene transfer ex vivo is considered the most versatile form of genetic medicine, as it can potentially treat the largest number of people and diseases. RNA modification therapies that target mRNA through approaches like antisense oligonucleotides, RNA interference, and ribozymes also show promise. While no genetic medicines have been approved yet to treat inherited disorders, areas like gene transfer, stem cell transplantation, RNA targeting therapies, and genome editing techniques continue to progress and hold hope to develop treatments for many conditions.

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd

    Question: Which of The Forms of Genetic Medicine Do You Think Is The M

    Uploaded by

    Mustafa Khudhair
    15 views1 page
    Gene therapy using viral vectors for gene transfer ex vivo is considered the most versatile form of genetic medicine, as it can potentially treat the largest number of people and diseases. RNA modification therapies that target mRNA through approaches like antisense oligonucleotides, RNA interference, and ribozymes also show promise. While no genetic medicines have been approved yet to treat inherited disorders, areas like gene transfer, stem cell transplantation, RNA targeting therapies, and genome editing techniques continue to progress and hold hope to develop treatments for many conditions.

    Original Description:

    Original Title

    -q46712173

    Copyright

    © © All Rights Reserved

    Available Formats

    PDF, TXT or read online from Scribd

    Did you find this document useful?

    Is this content inappropriate?

    Gene therapy using viral vectors for gene transfer ex vivo is considered the most versatile form of genetic medicine, as it can potentially treat the largest number of people and diseases. RNA modification therapies that target mRNA through approaches like antisense oligonucleotides, RNA interference, and ribozymes also show promise. While no genetic medicines have been approved yet to treat inherited disorders, areas like gene transfer, stem cell transplantation, RNA targeting therapies, and genome editing techniques continue to progress and hold hope to develop treatments for many conditions.

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd
    Download as pdf or txt
    15 views1 page

    Question: Which of The Forms of Genetic Medicine Do You Think Is The M

    Uploaded by

    Mustafa Khudhair
    Gene therapy using viral vectors for gene transfer ex vivo is considered the most versatile form of genetic medicine, as it can potentially treat the largest number of people and diseases. RNA modification therapies that target mRNA through approaches like antisense oligonucleotides, RNA interference, and ribozymes also show promise. While no genetic medicines have been approved yet to treat inherited disorders, areas like gene transfer, stem cell transplantation, RNA targeting therapies, and genome editing techniques continue to progress and hold hope to develop treatments for many conditions.

    Copyright:

    © All Rights Reserved
    Download as PDF, TXT or read online from Scribd
    Download as pdf or txt
    of 1

    home / study / science / biology / biology questions and answers / which of the forms of genetic medicine do you think

    is the most versatile (be a…

    Question: Which of the forms of genetic medicine do you think is the m…


    See this question in the app

    Which of the forms of genetic medicine do you think is the most versatile (be able to treat the largest
    number of people, the largest number of diseases, etc...)? Explain your reasoning.

    Expert Answer

    PRIYANKA BHARDWAJ answered this


    Was this answer helpful? 0 1
    80 answers

    Strategies for genetic medicines - that is ,therapies that use the transfer of DNA and /or RNA to modify
    gene expression to compensate for an abnormal phenotype - include the use of somatic stem cells ,gene
    transfer ,RNA modi cation and in the future ,embryonic stem cells .

    Bone marrow stem cell transplantation from individuals that express the normal gene has been used to
    treat various inherited diseases , including lysosomal storage disorders , immunode ciency,
    haemoglobinpathies and leukodystrophies.

    The main thrust in gene transfer strategies over the next several years will be able to devlop further
    :adeno associated vectors for invivo studies ,retrovirus vectors for ex vivo studies that involve autologous
    hametopietic stem cells .

    RNA modi cation therapy targets mRNA ,either to supress mRNA levels or by correcting or adding
    function to the m RNA using four basics approaches : antisense oligonucleotide,RNAi trans - splicing and
    ribozymes.

    The main biological barriers to all genetic medicines are delivery and maintenance of the new genetic
    information.Overcoming these hurdles requires an understanding of the : molecular basis of the disorder
    ,it's mode of inheritance,the range of mutations and genotype - phenotype relationship that result in the
    disease phenotype ,how the phenotye is modulated by alternative genes and how ,where and when the
    disease manifests .

    Gene transfer of the normal gene to an individual affected by a monogenic disorder is an obvious strategy
    for genetic medicine.

    Although many mouse models have been cured with gene transfer, in practice correcting human
    hereditary disorders has proved to be difficult.

    No genetic medicines has been approved for use in the treatment of any hereditary human disorder,but
    signi cant intellectual and economic resources are focused on genetic medicines.

    Antisense oligonucleotides (ASOs) are short strings of chemically modi ed DNA or RNA that are designed
    to home in on RNA strands to alter the proteins the body ultimately produces .

    After decades of struggling to get ASO s to work as drugs .

    Patients with a fatala version of diseases called spinal muscular atrophy are now surviving thanks to ASOs.

    Genome editing has extended our ability to elucidate the contribution of genetics to disease by
    promoting the verstile CRISPR / Cas 9 technology has been rapidlly extended.

    Comment

    You might also like