Category Archives: Gene therapy

Yoshiki Sasai is not just an ordinary tissue engineer who tries to coax stem cells to grow into fully formed bodily structures. It is true that Sasai has made his mark by taking on big projects like using stem cells to whip up a retina, cortical tissue and the cerebellum, involved with balance and movement. But his research has gone deeper by delving into the way stem cells organize themselves into complex structures under the influence of genes and the prenatal environment. Read a profile of Sasai here to accompany “ Grow Your Own Eye ,” Sasai’s own account of growing a retina in the November Scientific American .

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In the womb, a ball of identical cells gives rise to varied cell types that ultimately form highly ordered structures and then the full panoply of organs in the human body. The process advances according to an internal biological script that directs each fold and crease of tissue to assume exactly the proper shape and dimension.

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Disorders Of Hemoglobin - , Bernard G. Forget
ll4.me Disorders Of Hemoglobin - , Bernard G. Forget This book is a completely revised new edition of the definitive reference on disorders of hemoglobin. Authored by world-renowned experts, the book focuses on basic science aspects and clinical features of hemoglobinopathies, covering diagnosis, treatment, and future applications of current research. While the second edition continues to address the important molecular, cellular, and genetic components, coverage of clinical issues has been significantly expanded, and there is more practical emphasis on diagnosis and management throughout. The book opens with a review of the scientific underpinnings. Pathophysiology of common hemoglobin disorders is discussed next in an entirely new section devoted to vascular biology, the erythrocyte membrane, nitric oxide biology, and hemolysis. Four sections deal with #65533;? #65533; #65533;? #65533; and #65533;? #65533; thalassemia, sickle cell disease, and related conditions, followed by special topics. The second edition concludes with current and developing approaches to treatment, incorporating new agents for iron chelation, methods to induce fetal hemoglobin production, novel treatment approaches, stem cell transplantation, and progress in gene therapy.Author: Steinberg, Martin H. Author: Forget, Bernard G. Author: Higgs, Douglas R. Publisher: Cambridge University Press Illustration: N Language: ENG Title: Disorders of Hemoglobin Pages: 00848 (Encrypted PDF) On Sale: 2009-06-30 SKU-13/ISBN: 9780521875196 ...From:marylloyd321Views:0 0ratingsTime:00:13More inPeople Blogs

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Disorders Of Hemoglobin - , Bernard G. Forget - Video

Post-genomic Cardiology
ll4.me Post-genomic Cardiology Recent advances in molecular and cellular biology have markedly changed our understanding of the heart, and this is having tremendous ramifications for the clinician. This unique reference offers a comprehensive and critical evaluation of this contribution in the field of cardiovascular molecular medicine providing the reader with a sense of new directions in which molecular medicine might be applied. It begins with a detailed primer that makes readily accessible recent molecular, genetic and cellular techniques. Rounding out the coverage of this exciting field are critical and comprenhesive discussions on the use of molecular, genetic and cellular techniques used to identify the etiology and pathophysiology of specific cardiac diseases.* Discusses diagnostic and therapeutic options available not only in the adult and aging individuals but also in infants/children* Numerous illustrations and flow-charts* Explans cutting-edge molecular techniques, including analysis of mitochondria, their role in cardiac dysfunction and updated analysis of Cardioprotection and Metabolic Syndrome* Presentation of recent translational studies for the treatment of cardiovascular diseases is included (eg, gene therapy, pharmacological treatments and stem cell transplantation) Publisher: Academic Press Illustration: Y Language: ENG Title: Post-Genomic Cardiology Pages: 00000 (Encrypted PDF) On Sale: 2007-02-26 SKU-13/ISBN: 9780123736987 Category: Medical ...From:julianlewis9854Views:0 0ratingsTime:00:14More inPeople Blogs

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Post-genomic Cardiology - Video

Post-genomic Cardiology
ll4.me Post-genomic Cardiology Recent advances in molecular and cellular biology have markedly changed our understanding of the heart, and this is having tremendous ramifications for the clinician. This unique reference offers a comprehensive and critical evaluation of this contribution in the field of cardiovascular molecular medicine providing the reader with a sense of new directions in which molecular medicine might be applied. It begins with a detailed primer that makes readily accessible recent molecular, genetic and cellular techniques. Rounding out the coverage of this exciting field are critical and comprenhesive discussions on the use of molecular, genetic and cellular techniques used to identify the etiology and pathophysiology of specific cardiac diseases.* Discusses diagnostic and therapeutic options available not only in the adult and aging individuals but also in infants/children* Numerous illustrations and flow-charts* Explans cutting-edge molecular techniques, including analysis of mitochondria, their role in cardiac dysfunction and updated analysis of Cardioprotection and Metabolic Syndrome* Presentation of recent translational studies for the treatment of cardiovascular diseases is included (eg, gene therapy, pharmacological treatments and stem cell transplantation) Publisher: Academic Press Illustration: Y Language: ENG Title: Post-Genomic Cardiology Pages: 00000 (Encrypted PDF) On Sale: 2007-02-26 SKU-13/ISBN: 9780123736987 Category: Medical ...From:julianlewis9854Views:0 0ratingsTime:00:14More inPeople Blogs

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Post-genomic Cardiology - Video

By Chris Reidy, Globe Staff

bluebird bio, a Cambridge company that eschews capital letters, said in a Friday press release that the California Institute for Regenerative Medicine has approved a $9.3 million award to support the development one of bluebird bios gene therapies.

The award will support the testing of a gene therapy called LentiGlobin, said the company, which also has an office in San Francisco.

LentiGlobin is designed to treat beta-thalassemia, an inherited blood disorder that causes the body to have an inadequate amount of functional hemoglobin; in its most severe form, patients typically require life-long monthly supportive red blood cell transfusions to treat their severe anemia, according to bluebird bios website.

According to bluebird bio, LentiGlobin is a one-time transformative gene therapy for patients with beta-thalassemia.

The California Institute for Regenerative Medicine was established in November 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for stem cell research.

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bluebird bio is awarded $9.3m to support its gene therapy treatments

ScienceDaily (Oct. 24, 2012) Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

The research results are online Oct. 24, in the journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24

The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.

The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.

"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."

To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.

"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study were allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."

The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.

The second portion of the study, which was completed at ONPRC, is also considered an important achievement because egg cells only remain viable for a short period of time after they are harvested from a donor. Therefore, for this therapy to be a viable option in the clinic, preservation through freezing likely is necessary so that both the donor cell and a mother's cell are viable at the time of the procedure.

While this form of therapy has yet to be approved in the United States, the United Kingdom is seriously considering its use for treating human patients at risk for mitochondria-based disease. It's believed that this most recent breakthrough, combined with earlier animal studies, will help inform that decision-making process.

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New gene therapy method tested in human cells ... and it works, researchers report

Public release date: 24-Oct-2012 [ | E-mail | Share ]

Contact: Jim Newman newmanj@ohsu.edu 503-494-8231 Oregon Health & Science University

PORTLAND, Ore. - Oregon Health & Science University's development of a new gene therapy method to prevent certain inherited diseases has reached a significant milestone. Researchers at the university's Oregon National Primate Research Center and the OHSU Department of Obstetrics & Gynecology have successfully demonstrated their procedure in human cells. It's believed that this research, along with other efforts, will pave the way for future clinical trials in human subjects.

The research results are online Wednesday, Oct. 24, in the highly respected journal Nature. Dr. Mitalipov also will present the results of his research at the American Society for Reproductive Medicine Conference in San Diego Oct. 24'.

The OHSU gene therapy method was initially devised through research in nonhuman primates led by Shoukhrat Mitalipov, Ph.D., associate scientist in the Division of Reproductive & Developmental Sciences at ONPRC, Oregon Stem Cell Center and OHSU School of Medicine departments of Obstetrics and Gynecology and Molecular and Medical Genetics.

The procedure was specifically developed to prevent diseases related to gene defects in the cell mitochondria. Mitalipov's previous work was published in the August 2009 edition of Nature. In the current study, Mitalipov, in collaboration with Paula Amato, M.D., associate professor of obstetrics and gynecology in the OHSU Center for Women's Health, demonstrated efficacy of this therapy in human gametes and embryos.

"Cell mitochondria contain genetic material just like the cell nucleus and these genes are passed from mother to infant," explained Mitalipov. "When certain mutations in mitochondrial DNA are present, a child can be born with severe conditions, including diabetes, deafness, eye disorders, gastrointestinal disorders, heart disease, dementia and several other neurological diseases. Because mitochondrial-based genetic diseases are passed from one generation to the next, the risk of disease is often quite clear. The goal of this research is to develop a therapy to prevent transmission of these disease-causing gene mutations."

To conduct this research, Mitalipov and his colleagues obtained 106 human egg cells from study volunteers recruited through OHSU's Division of Fertility and Reproductive Endocrinology. The researchers then used a method developed in previous nonhuman primate studies, to transfer the nucleus from one cell to another. In effect, the researchers "swapped out" the cell cytoplasm, which contains the mitochondria. The egg cells were then fertilized to determine whether the transfer was a success and whether the cells developed normally. Upon inspection, it was demonstrated that it was possible to successfully replace mitochondrial DNA using this method.

"Using this process, we have shown that mutated DNA from the mitochondria can be replaced with healthy copies in human cells," explained Mitalipov. "While the human cells in our study only allowed to develop to the embryonic stem cell stage, this research shows that this gene therapy method may well be a viable alternative for preventing devastating diseases passed from mother to infant."

The current Nature paper also expanded upon the previously reported nonhuman primate work by demonstrating that the method was possible using frozen egg cells. Mitochondria were replaced in a frozen/thawed monkey egg cell, resulting in the birth of a healthy baby monkey named Chrysta.

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OHSU researchers test new gene therapy method in human cells... and it works

The Press Release from the Nobel Assembly at Karolinska Institute

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The Press Release from the Nobel Assembly at Karolinska Institute

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