Gene therapy for sickle cell disease

Abstract

Cochrane Database of Systematic Reviews Gene therapy for sickle cell disease (Review) Olowoyeye A, Okwundu CI Olowoyeye A, Okwundu CI. Gene therapy for sickle cell disease.

Oligonucleotide-Recognizing Topoisomerase Inhibitors (OTIs): Precision Gene Editors for Neurodegenerative Diseases?

“… biophysical assays (such as swithSENSE-[ 98 ]) to optimize relative affinities of modified OTIs for target DNA sequences ‘in vitro’-with purified topoisomerases. using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

“…using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

Oligonucleotide-Recognizing Topoisomerase Inhibitors (OTIs): Precision Gene Editors for Neurodegenerative Diseases?

“… biophysical assays (such as swithSENSE-[ 98 ]) to optimize relative affinities of modified OTIs for target DNA sequences ‘in vitro’-with purified topoisomerases. using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

“…using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

Intervertebral Disc Diseases PART 2: A Review of the Current Diagnostic and Treatment Strategies for Intervertebral Disc Disease

“…So far, gene therapy applications are limited to medically life-threatening conditions, such as cystic fibrosis and sickle cell disease, rather than degenerative conditions. The use of non-viral vectors is not as reliable in genetic transfection [108][109][110][111]. These are current challenges limiting the amount of research in gene therapy.…”

Anemia falciforme y la resistencia a la malaria. Revisión narrativa

“…Por último, como terapia curativa, el trasplante de células madres hematopoyéticas es la única medida aprobada con este fin, pero existen múltiples barreras para su implementación como la falta de donantes compatibles, el rechazo del trasplante y los desenlaces adversos o inciertos a largo plazo (45). La terapia génica es una alternativa curativa de la enfermedad (48); en la anemia falciforme busca reemplazar el gen de la hemoglobina defectuoso con una copia normal de ese gen, para así restaurar la función de la proteína de forma definitiva (49). La terapia génica aún no está disponible en nuestro medio por lo que el trasplante continúa siendo la única terapia curativa a la que se tiene acceso.…”

Oligonucleotide-Recognizing Topoisomerase Inhibitors (OTIs): Precision Gene Editors for Neurodegenerative Diseases?

“… biophysical assays (such as swithSENSE-[ 98 ]) to optimize relative affinities of modified OTIs for target DNA sequences ‘in vitro’-with purified topoisomerases. using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

“…using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

Intervertebral Disc Diseases PART 2: A Review of the Current Diagnostic and Treatment Strategies for Intervertebral Disc Disease

“…So far, gene therapy applications are limited to medically life-threatening conditions, such as cystic fibrosis and sickle cell disease, rather than degenerative conditions. The use of non-viral vectors is not as reliable in genetic transfection [108][109][110][111]. These are current challenges limiting the amount of research in gene therapy.…”

Anemia falciforme y la resistencia a la malaria. Revisión narrativa

“…Por último, como terapia curativa, el trasplante de células madres hematopoyéticas es la única medida aprobada con este fin, pero existen múltiples barreras para su implementación como la falta de donantes compatibles, el rechazo del trasplante y los desenlaces adversos o inciertos a largo plazo (45). La terapia génica es una alternativa curativa de la enfermedad (48); en la anemia falciforme busca reemplazar el gen de la hemoglobina defectuoso con una copia normal de ese gen, para así restaurar la función de la proteína de forma definitiva (49). La terapia génica aún no está disponible en nuestro medio por lo que el trasplante continúa siendo la única terapia curativa a la que se tiene acceso.…”

Oligonucleotide-Recognizing Topoisomerase Inhibitors (OTIs): Precision Gene Editors for Neurodegenerative Diseases?

“… biophysical assays (such as swithSENSE-[ 98 ]) to optimize relative affinities of modified OTIs for target DNA sequences ‘in vitro’-with purified topoisomerases. using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

“…using nuclease resistant OTIs in iPSC cells to compare their gene editing functionality with that of published CRISPR/Cas systems for similar diseases (e.g., [ 97 , 99 , 100 ]). We assume initially that OTI development pathways would aim at replicating and improving on existing CRISPR/Cas gene editing in iPSCs.…”

Intervertebral Disc Diseases PART 2: A Review of the Current Diagnostic and Treatment Strategies for Intervertebral Disc Disease

“…So far, gene therapy applications are limited to medically life-threatening conditions, such as cystic fibrosis and sickle cell disease, rather than degenerative conditions. The use of non-viral vectors is not as reliable in genetic transfection [108][109][110][111]. These are current challenges limiting the amount of research in gene therapy.…”

Anemia falciforme y la resistencia a la malaria. Revisión narrativa

“…Por último, como terapia curativa, el trasplante de células madres hematopoyéticas es la única medida aprobada con este fin, pero existen múltiples barreras para su implementación como la falta de donantes compatibles, el rechazo del trasplante y los desenlaces adversos o inciertos a largo plazo (45). La terapia génica es una alternativa curativa de la enfermedad (48); en la anemia falciforme busca reemplazar el gen de la hemoglobina defectuoso con una copia normal de ese gen, para así restaurar la función de la proteína de forma definitiva (49). La terapia génica aún no está disponible en nuestro medio por lo que el trasplante continúa siendo la única terapia curativa a la que se tiene acceso.…”