Pub Date : 2014-02-01DOI: 10.4172/2169-0111.1000E108
Youming Zhang
With the rapid development of zinc finger nucleases (ZFN), transcription activator-like effector nuclease (TALEN) and Cluster regularly interspaced short palindromic repeat (CRISPR)/Cas systems, manipulating genome becomes relatively easy and efficient. There are two major purposes for genome manipulation: one is for understanding novel genes function and their regulation roles in cells.Recent genomic and genetic approaches for disease genes have identified many novel loci underlie the disease phenotypes. More than 2,000 robust associations have been found in more than 300 complex diseases and traitsin recent years by genome wide association studies (GWASs) [1]. Understudying the function roles of genetic loci, particular novel genes will bring new insight of the diseases mechanisms. The second major application for manipulating genome is providing a new therapeutic means for many genetic disorders.A disease causing mutation could be replaced with normal allele in several Mendelian monogenic diseases.
{"title":"Genome Editing with ZFN, TALEN and CRISPR/Cas Systems:The Applications and Future Prospects","authors":"Youming Zhang","doi":"10.4172/2169-0111.1000E108","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E108","url":null,"abstract":"With the rapid development of zinc finger nucleases (ZFN), transcription activator-like effector nuclease (TALEN) and Cluster regularly interspaced short palindromic repeat (CRISPR)/Cas systems, manipulating genome becomes relatively easy and efficient. There are two major purposes for genome manipulation: one is for understanding novel genes function and their regulation roles in cells.Recent genomic and genetic approaches for disease genes have identified many novel loci underlie the disease phenotypes. More than 2,000 robust associations have been found in more than 300 complex diseases and traitsin recent years by genome wide association studies (GWASs) [1]. Understudying the function roles of genetic loci, particular novel genes will bring new insight of the diseases mechanisms. The second major application for manipulating genome is providing a new therapeutic means for many genetic disorders.A disease causing mutation could be replaced with normal allele in several Mendelian monogenic diseases.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"3 1","pages":"1-3"},"PeriodicalIF":0.0,"publicationDate":"2014-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4172/2169-0111.1000E108","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877150","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2014-01-02DOI: 10.4172/2169-0111.1000E107
A. Chhabra
Targeted genome editing is essential for functional characterization of a gene of interest. Targeted gene inactivation via homologous recombination made it feasible to create gene knockout animal models to ascertain the physiological role of the target genes; however, lower efficiency of site specific insertion of the genetically modified construct through homologous recombination has limited a wider applicability of this approach. Development of targeted gene knockdown through RNA interferce (RNAi) offered a cost effective, high-throughput alternative to homologous recombination, however, RNAi-mediated gene knockdown is incomplete, produces experiment to experiment variation, and could provide only a temporary inhibition of the gene function. Development of genome engineering methodologies utilizing nucleases linked to the guide sequences targeting a gene of interest, such as Zinc Finger Nucleases (ZFN), Transcription Activator like Effector Nucleases (TALEN) and Clustered Palindromic Repeats (CRISPR), are quite encouraging. A brief overview of recent advances in genome engineering approaches is provided with their respective advantages and limitations.
{"title":"Advances in Genome Engineering Approaches","authors":"A. Chhabra","doi":"10.4172/2169-0111.1000E107","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E107","url":null,"abstract":"Targeted genome editing is essential for functional characterization of a gene of interest. Targeted gene inactivation via homologous recombination made it feasible to create gene knockout animal models to ascertain the physiological role of the target genes; however, lower efficiency of site specific insertion of the genetically modified construct through homologous recombination has limited a wider applicability of this approach. Development of targeted gene knockdown through RNA interferce (RNAi) offered a cost effective, high-throughput alternative to homologous recombination, however, RNAi-mediated gene knockdown is incomplete, produces experiment to experiment variation, and could provide only a temporary inhibition of the gene function. Development of genome engineering methodologies utilizing nucleases linked to the guide sequences targeting a gene of interest, such as Zinc Finger Nucleases (ZFN), Transcription Activator like Effector Nucleases (TALEN) and Clustered Palindromic Repeats (CRISPR), are quite encouraging. A brief overview of recent advances in genome engineering approaches is provided with their respective advantages and limitations.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"3 1","pages":"1-4"},"PeriodicalIF":0.0,"publicationDate":"2014-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4172/2169-0111.1000E107","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877093","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2014-01-01DOI: 10.4172/2169-0111.1000E106
K. Hefferon
established that a large number of infectious diseases utilize the mucosal membrane within the gut to enter the body; therefore vaccines which can be expressed within edible plant tissue offer a select advantage. As a result, plant cells not only act as the delivery vehicle itself, they protect the antigen as it passes through the harsh environment of the gastrointestinal tract. The fact that plants can produce recombinant antigens that undergo post-translational modifications which are largely similar to their mammalian-derived counterparts provides
{"title":"The Potential Impact of Plant-made Biopharmaceuticals for the Improvement of Global Health","authors":"K. Hefferon","doi":"10.4172/2169-0111.1000E106","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E106","url":null,"abstract":"established that a large number of infectious diseases utilize the mucosal membrane within the gut to enter the body; therefore vaccines which can be expressed within edible plant tissue offer a select advantage. As a result, plant cells not only act as the delivery vehicle itself, they protect the antigen as it passes through the harsh environment of the gastrointestinal tract. The fact that plants can produce recombinant antigens that undergo post-translational modifications which are largely similar to their mammalian-derived counterparts provides","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"3 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2014-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877076","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2014-01-01DOI: 10.4172/2169-0111.1000E105
Z. Jaradat
Previously the concept of genetics was applied to a limited number of diseases with obvious phenotypes where simple people use to observe and relate. It was never easy to establish a clear cut of heritability range between genetic variation and a risk of suffering from a complex disorder due to many reasons including but not limited to; gene variations, non-linear interactions between genetic variance and phenotype severity, complex gene-gene interactions and many others [1]. However, every day, scientists in a research lab somewhere discover a genetic linkage to a disease or a syndrome. This has gone far beyond simple imagination where traits or syndromes used to be related almost exclusively to environment are now genetically linked. The difficulty of identification of patterns among the affected people and the involvement of multiple genes and environmental effects were all behind the difficulty in understanding the link of some of these syndromes/diseases or desired traits to genetics. Advances in genetics/ genomics and molecular biology have enabled geneticists to decipher some of the genes that are believed to be linked to some diseases. Recently genetic links has been established to many entities some of which were never thought of as linked with genetics while some were linked to genetics but with no understanding of their patterns. Obesity, neurological disorders, intelligence, asthma, cardiovascular diseases, periodontal diseases, longevity, deafness, cataracts, cancer are just few of the entities that are positively linked to genetics. This editorial will focus mainly on neuropsychiatric genetics including substance addiction, smoking and the genetics of anger.
{"title":"Genetics Beyond Diabetes and Baldness","authors":"Z. Jaradat","doi":"10.4172/2169-0111.1000E105","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E105","url":null,"abstract":"Previously the concept of genetics was applied to a limited number of diseases with obvious phenotypes where simple people use to observe and relate. It was never easy to establish a clear cut of heritability range between genetic variation and a risk of suffering from a complex disorder due to many reasons including but not limited to; gene variations, non-linear interactions between genetic variance and phenotype severity, complex gene-gene interactions and many others [1]. However, every day, scientists in a research lab somewhere discover a genetic linkage to a disease or a syndrome. This has gone far beyond simple imagination where traits or syndromes used to be related almost exclusively to environment are now genetically linked. The difficulty of identification of patterns among the affected people and the involvement of multiple genes and environmental effects were all behind the difficulty in understanding the link of some of these syndromes/diseases or desired traits to genetics. Advances in genetics/ genomics and molecular biology have enabled geneticists to decipher some of the genes that are believed to be linked to some diseases. Recently genetic links has been established to many entities some of which were never thought of as linked with genetics while some were linked to genetics but with no understanding of their patterns. Obesity, neurological disorders, intelligence, asthma, cardiovascular diseases, periodontal diseases, longevity, deafness, cataracts, cancer are just few of the entities that are positively linked to genetics. This editorial will focus mainly on neuropsychiatric genetics including substance addiction, smoking and the genetics of anger.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"3 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2014-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.4172/2169-0111.1000E105","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877469","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-11-28DOI: 10.4172/2169-0111.1000E104
M. Conese
{"title":"Gene Therapy Gets its Momentum through the Marketing of an Engineered Virus to Treat Lipoprotein Lipase Deficiency","authors":"M. Conese","doi":"10.4172/2169-0111.1000E104","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E104","url":null,"abstract":"","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"2 1","pages":"1-3"},"PeriodicalIF":0.0,"publicationDate":"2013-11-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-11-15DOI: 10.4172/2169-0111.1000116
A. Mustafayeva, V. Mammadov, I. Ahmadov, L. Jafarova
The research focused on legal regulation of spread, import and production of GMOs, analyzing GMO issue as new and important for Azerbaijan. This problem is topical for the Azerbaijani public and scientific society as well as the state governance. GMO issues reflected in the laws of Azerbaijan have been studied and compared with the laws of other countries, in particular, CIS countries. As a result, the authors made certain suggestions to include GMO issues in Azerbaijan laws, even to pass a separate law
{"title":"Legal Regulation of Genetically Modified Products in Azerbaijan","authors":"A. Mustafayeva, V. Mammadov, I. Ahmadov, L. Jafarova","doi":"10.4172/2169-0111.1000116","DOIUrl":"https://doi.org/10.4172/2169-0111.1000116","url":null,"abstract":"The research focused on legal regulation of spread, import and production of GMOs, analyzing GMO issue as new and important for Azerbaijan. This problem is topical for the Azerbaijani public and scientific society as well as the state governance. GMO issues reflected in the laws of Azerbaijan have been studied and compared with the laws of other countries, in particular, CIS countries. As a result, the authors made certain suggestions to include GMO issues in Azerbaijan laws, even to pass a separate law","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"2 1","pages":"1-5"},"PeriodicalIF":0.0,"publicationDate":"2013-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70874552","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-11-06DOI: 10.4172/2169-0111.1000E103
Yongmin Yan, D. Wei
In this post-genome era of advanced high-throughput DNA/RNA sequencing technologies, information may no longer be a bottleneck to understand and tackle complicated genetic diseases such as cancer. What is still lacking, however, is an efficient, reliable, and easy tool to precisely modify the cellular genome for functional genome annotation, disease modeling, and possibly even corrective gene therapy.
{"title":"The CRISPR-Cas9 System: A Powerful Tool for Genome Engineering and Regulation","authors":"Yongmin Yan, D. Wei","doi":"10.4172/2169-0111.1000E103","DOIUrl":"https://doi.org/10.4172/2169-0111.1000E103","url":null,"abstract":"In this post-genome era of advanced high-throughput DNA/RNA sequencing technologies, information may no longer be a bottleneck to understand and tackle complicated genetic diseases such as cancer. What is still lacking, however, is an efficient, reliable, and easy tool to precisely modify the cellular genome for functional genome annotation, disease modeling, and possibly even corrective gene therapy.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"2 1","pages":"1-2"},"PeriodicalIF":0.0,"publicationDate":"2013-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877332","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-10-30DOI: 10.4172/2169-0111.S1.002
Tian-wen He
Natural products, particularly polyketides are among the most important sources of antimicrobial compounds. 20% of the top selling drugs are polyketide based. In recent years genetic engineering has played a critical role in modifying biosynthetic pathways of different polyketide compounds as a way to create novel structures with improved clinical properties. Further investigation and understanding of these giant multi-enzyme complexes is necessary to achieve efficient synthetic engineering. In many PKS systems including the mupirocin biosynthesis pathway, the thioesterase (TE) is normally considered as the end of the assembly line. However, expressing the CoA-ligase tmlU from the thiomarinol pathway in the mupirocin producer strain (Pseudomonas fluorescens NCIMB10586) revealed that TmlU could only release truncated pseudomonic acid when a TE domain was present. This finding led to the hypothesis that perhaps the TE domain could act as a tether for TmlU, in order for the latter to be able to capture the growing chain and perhaps load it onto the post TE pathway. This study also presents the first evidence of MmpB being involved in producing the 9-hydroxynonanoic acid in the mupirocin biosynthesis pathway.
{"title":"HLA-A24 is an unfavorable clinical prognostic factor in patients with stage III~IV (advanced) nasopharyngeal carcinoma","authors":"Tian-wen He","doi":"10.4172/2169-0111.S1.002","DOIUrl":"https://doi.org/10.4172/2169-0111.S1.002","url":null,"abstract":"Natural products, particularly polyketides are among the most important sources of antimicrobial compounds. 20% of the top selling drugs are polyketide based. In recent years genetic engineering has played a critical role in modifying biosynthetic pathways of different polyketide compounds as a way to create novel structures with improved clinical properties. Further investigation and understanding of these giant multi-enzyme complexes is necessary to achieve efficient synthetic engineering. \u0000 \u0000In many PKS systems including the mupirocin biosynthesis pathway, the thioesterase (TE) is normally considered as the end of the assembly line. However, expressing the CoA-ligase tmlU from the thiomarinol pathway in the mupirocin producer strain (Pseudomonas fluorescens NCIMB10586) revealed that TmlU could only release truncated pseudomonic acid when a TE domain was present. This finding led to the hypothesis that perhaps the TE domain could act as a tether for TmlU, in order for the latter to be able to capture the growing chain and perhaps load it onto the post TE pathway. This study also presents the first evidence of MmpB being involved in producing the 9-hydroxynonanoic acid in the mupirocin biosynthesis pathway.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2013-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877974","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-10-30DOI: 10.4172/2169-0111.S1.001
T. Bera
T therapy of cancer is getting more traction over the traditional chemotherapy to overcome the unavoidable side effects from the chemotherapeutic drugs. As scientists began to understand more about the molecular mechanism that distinguishes the cancer cells from its normal counterpart, they began to design new approach to attack the cancer cells more precisely. One of such successful approach is the development of monoclonal antibody against a cancer specific surface antigen that can be used as a delivery vehicle of toxic material selectively to the cancer cells. Our laboratory is involved in generating recombinant immunotoxin (RIT) as targeted therapeutic protein to treat cancer. Recombinant immunotoxins are chimeric proteins composed of the Fv portion of an antibody fused to a portion a lethal toxin. The immunotoxin binds to a surface antigen on a cancer cells, enters the cell by endocytosis where it inactivate the cellular machinery and eventually kills the cell. These proteins are expressed in E. coli, purified as clinical grade material and been tested in patients with various types of cancer in clinical trials. Modern molecular biological techniques were used to improve the efficacy of these agents in vitro and test them in the preclinical model system. Many immunotoxins have been tested in clinical trials but most success has been achieved in patients with hematologic tumors. Poor response against solid tumor for these agents is likely due to poor penetration into tumor masses as well as the neutralizing immune response to the toxin component of the immunotoxin. Studies are in progress in the laboratory to overcome those issues and eventually test them in clinical trials.
{"title":"Recombinant immunotoxin as targeted therapeutic protein for cancer therapy","authors":"T. Bera","doi":"10.4172/2169-0111.S1.001","DOIUrl":"https://doi.org/10.4172/2169-0111.S1.001","url":null,"abstract":"T therapy of cancer is getting more traction over the traditional chemotherapy to overcome the unavoidable side effects from the chemotherapeutic drugs. As scientists began to understand more about the molecular mechanism that distinguishes the cancer cells from its normal counterpart, they began to design new approach to attack the cancer cells more precisely. One of such successful approach is the development of monoclonal antibody against a cancer specific surface antigen that can be used as a delivery vehicle of toxic material selectively to the cancer cells. Our laboratory is involved in generating recombinant immunotoxin (RIT) as targeted therapeutic protein to treat cancer. Recombinant immunotoxins are chimeric proteins composed of the Fv portion of an antibody fused to a portion a lethal toxin. The immunotoxin binds to a surface antigen on a cancer cells, enters the cell by endocytosis where it inactivate the cellular machinery and eventually kills the cell. These proteins are expressed in E. coli, purified as clinical grade material and been tested in patients with various types of cancer in clinical trials. Modern molecular biological techniques were used to improve the efficacy of these agents in vitro and test them in the preclinical model system. Many immunotoxins have been tested in clinical trials but most success has been achieved in patients with hematologic tumors. Poor response against solid tumor for these agents is likely due to poor penetration into tumor masses as well as the neutralizing immune response to the toxin component of the immunotoxin. Studies are in progress in the laboratory to overcome those issues and eventually test them in clinical trials.","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2013-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2013-10-30DOI: 10.4172/2169-0111.S1.003
Y. Kazuki
Luz M. Lopez-Marin received her M.Sc. and Ph.D. in Biochemistry from the Paul Sabatier University, Toulouse, France. She has been studying the immune response to infectious diseases at the National University of Mexico (UNAM) since 1994 and joined the Center of Applied Physics and Advanced Technology, within the same university, in 2010. She has published more than 25 papers in reputed journals. Lipoplexes and shock waves: A promising strategy for gene therapy
{"title":"Mouse artificial chromosome vectors for animal transgenesis","authors":"Y. Kazuki","doi":"10.4172/2169-0111.S1.003","DOIUrl":"https://doi.org/10.4172/2169-0111.S1.003","url":null,"abstract":"Luz M. Lopez-Marin received her M.Sc. and Ph.D. in Biochemistry from the Paul Sabatier University, Toulouse, France. She has been studying the immune response to infectious diseases at the National University of Mexico (UNAM) since 1994 and joined the Center of Applied Physics and Advanced Technology, within the same university, in 2010. She has published more than 25 papers in reputed journals. Lipoplexes and shock waves: A promising strategy for gene therapy","PeriodicalId":89733,"journal":{"name":"Advancements in genetic engineering","volume":"1 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2013-10-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"70877652","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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